#  @medstudentinvst WaveStrum Life Sciences WaveStrum Life Sciences posts on X about $prme, $ntla, $pepg, in the the most. They currently have [-----] followers and [---] posts still getting attention that total [---] engagements in the last [--] hours. ### Engagements: [---] [#](/creator/twitter::1948243155779792896/interactions)  - [--] Week [-----] -46% - [--] Month [------] -13% - [--] Months [-------] +333% - [--] Year [---------] +11,724% ### Mentions: [--] [#](/creator/twitter::1948243155779792896/posts_active)  - [--] Week [--] +41% - [--] Month [--] -7.50% - [--] Months [---] +2,777% - [--] Year [---] +37,500% ### Followers: [-----] [#](/creator/twitter::1948243155779792896/followers)  - [--] Week [-----] +1.30% - [--] Month [-----] +3.60% - [--] Months [-----] +2,793% ### CreatorRank: [---------] [#](/creator/twitter::1948243155779792896/influencer_rank)  ### Social Influence **Social category influence** [stocks](/list/stocks) [finance](/list/finance) [cryptocurrencies](/list/cryptocurrencies) [technology brands](/list/technology-brands) [countries](/list/countries) [currencies](/list/currencies) [travel destinations](/list/travel-destinations) **Social topic influence** [$prme](/topic/$prme) #14, [$ntla](/topic/$ntla), [$pepg](/topic/$pepg) #7, [in the](/topic/in-the), [update](/topic/update), [daily](/topic/daily), [$nktr](/topic/$nktr) #25, [$qure](/topic/$qure), [$crsp](/topic/$crsp), [$pasg](/topic/$pasg) #7 **Top accounts mentioned or mentioned by** [@geneinvesting](/creator/undefined) [@seedy19tron](/creator/undefined) [@zhaoweiasu](/creator/undefined) [@endpts](/creator/undefined) [@martinshkreli](/creator/undefined) [@drjesstaylor](/creator/undefined) [@plainyogurt21](/creator/undefined) [@aditharun](/creator/undefined) [@grok](/creator/undefined) [@amaymd](/creator/undefined) [@capitalshipyard](/creator/undefined) [@winklevosscap](/creator/undefined) [@biopharmiq](/creator/undefined) [@melvinriskmgmt](/creator/undefined) [@cdcgov](/creator/undefined) [@capitalusher](/creator/undefined) [@stmkrs](/creator/undefined) [@cernovich](/creator/undefined) [@monacobiotech](/creator/undefined) [@hall8jack](/creator/undefined) **Top assets mentioned** [Prime Medicine, Inc. (PRME)](/topic/$prme) [Intellia Therapeutics, Inc (NTLA)](/topic/$ntla) [PepGen Inc. (PEPG)](/topic/$pepg) [uniQure N.V. (QURE)](/topic/$qure) [CRISPR Therapeutics AG (CRSP)](/topic/$crsp) [Passage (PASG)](/topic/$pasg) [Abivax SA (ABVX)](/topic/$abvx) [SuperRare (RARE)](/topic/$rare) [Vaxcyte, Inc. (PCVX)](/topic/$pcvx) [Avidity Biosciences, Inc. (RNA)](/topic/$rna) [Alnylam Pharmaceuticals, Inc. (ALNY)](/topic/$alny) [BEAM (BEAM)](/topic/$beam) [Wave Life Sciences Ltd. Ordinary Shares (WVE)](/topic/$wve) [Arrowhead Research Corporation (ARWR)](/topic/$arwr) [BridgeBio Pharma, Inc. Common Stock (BBIO)](/topic/$bbio) [Eli Lilly and Company (LLY)](/topic/$lly) [Janux Therapeutics, Inc. (JANX)](/topic/$janx) [Verve Therapeutics Inc. (VERV)](/topic/$verv) [Viking Therapeutics, Inc (VKTX)](/topic/$vktx) [The Pink Panther (PRIME)](/topic/$prime) [Viking Holdings Ltd (VIK)](/topic/$vik) [Metagenomi Technologies, LLC (MGX)](/topic/$mgx) [NVIDIA Corp. (NVDA)](/topic/$nvda) ### Top Social Posts Top posts by engagements in the last [--] hours "$BBC's decline of -43.43% since August [----] relative to $SPY's gain of +90.91% shows just how poorly the biotech sector has performed in terms of valuations and underlying capital outflows. This has occurred amidst massive upwards pressure on equity values from inflation in general (+24ish% since 8/2020) and enormous M2 expansion in specific (up more than 40% from the start of 2020) tempered marginally by a rising rate environment. Significant renewed interest in the biotech sector and significant renewed capital inflows from non-life science asset managers prospective life science LPs and" [X Link](https://x.com/medstudentinvst/status/1955688241299902663) 2025-08-13T17:49Z [--] followers, [--] engagements "$PEPG 13F Updates Q2 [----] Top [--] Investors (45.5% of shares outstanding): [--]. RA Capital: [--------] shares (HOLD Q1 to Q2 '25) [--]. Point72: [-------] shares (3.1% INCREASE in Q2 '25 from [-------] in Q1 '25) [--]. Viking Global: [-------] shares (HOLD Q1 to Q2 '25)" [X Link](https://x.com/medstudentinvst/status/1956100407387632068) 2025-08-14T21:07Z [--] followers, [---] engagements "$PEPG 13F Updates Q2 [----] Top [--] Institutional Investors (45.5% of shares outstanding): [--]. RA Capital: [--------] shares (HOLD Q1 to Q2 '25) [--]. Point72: [-------] shares (3.1% INCREASE in Q2 '25 from [-------] in Q1 '25) [--]. Viking Global: [-------] shares (HOLD Q1 to Q2 '25)" [X Link](https://x.com/medstudentinvst/status/1956101843836395639) 2025-08-14T21:13Z [---] followers, [---] engagements "August 15th [----]. Small adds to $RARE and $NTLA today and doubled the size of $NKTR position. $NTLA position getting very close to maximum sizing I am comfortable with. $RARE starting to get close as well. A substantial percentage of available capital has now been deployed. This fall will have a lot of key data readouts for the portfolio. Key task this weekend is to make a decision on whether to hold or sell $ALNY" [X Link](https://x.com/anyuser/status/1956566509616005258) 2025-08-16T03:59Z [----] followers, [----] engagements "For whatever it is worth the enrollment rates in nex-z's Phase III trial is the best empirical evidence we have regarding the degree of patient and physician demand for nex-z especially as enrollment is occurring at the same time as acoramidis's and vutrisiran's commercial ramp and nucresiran entering Phase III. The fact $NTLA expects to enroll [---] patients by year end (first dose in 2024) is a reasonable indication demand exists. Just [---] patients a year in commercial stage would almost certainly get $NTLA to at least a $1.77Bn EV from its current $1.18Bn (conservatively: $1.5M price *4.5x" [X Link](https://x.com/medstudentinvst/status/1957145864654184916) 2025-08-17T18:21Z [--] followers, [--] engagements "You are missing the critical statistic. What percent of the aggregated ceiling value of the contracts in each size bucket is spent Or provide a bar graph showing the percentage of contracts within different buckets of percentage of ceiling value spent. If 38% of contracts over $1Bn were spent to ceiling and 61% of $1Bn+ contracts were spent to say 98% of ceiling value your graph would presumably still just show that 38% even though 99% of all $1Bn contracts are effectively spent to ceiling. That would be highly misleading. I dont know what percentage of aggregated ceiling contract value gets" [X Link](https://x.com/medstudentinvst/status/1957405257295610138) 2025-08-18T11:32Z [--] followers, [--] engagements "$CRSPR in vivo is basically just first-gen CRISPR's version of $VERV with a dash of a permanent version of $ALNY / $ARWR cardiovascular and AHP. Will say Lp(a) is a massive opportunity that I don't think is fully appreciated. Population wide testing and treatment should become standard of care within the next decade and will produce at least one blockbuster maybe more. [--]. 20%-30% of population have elevated levels [--]. Biggest inheritable risk factor for CVD which is the leading cause of death in the US and globally [--]. Risk is cumulative and driven by number of years of exposure so early" [X Link](https://x.com/medstudentinvst/status/1957451318957670591) 2025-08-18T14:35Z [--] followers, [---] engagements "Prime Medicine - $PRIME. Potential to cure (not just treat) 90% of genetic diseases and transform medicine in unprecedented way. Somewhat derisked as $VERV bought out for $1Bn and has MC of $471M. Nobel Prize level science. Technology out of Broad / Harvard / MIT. Alphabet largest institutional investor" [X Link](https://x.com/medstudentinvst/status/1957577488999281019) 2025-08-18T22:56Z [--] followers, [--] engagements "The answer is compound growth. $PRME is effectively a venture play in a company that happens to be publicly traded. Lets say $PRIME becomes a $50Bn company if successful. I think anyone who is an investor at this stage has to be thinking $PRMEs upside case is at minimum near $ALNYs valuation. That would be about 100x from current prices. Investing now at $3.50 vs $7.00 a year down the line may not seem like much of a difference. However that would halve a 100x return to 50x. Similarly investing at $1.75 a few weeks ago would have made the return 200x at the same $50Bn EV. Thats a ton of" [X Link](https://x.com/anyuser/status/1957651003697729912) 2025-08-19T03:49Z [----] followers, [----] engagements "Yes its certainly true if $NTLA goes up 4x in the timeframe it takes $PRME to go up 3x one can get an additional 1x of return to invest back to $PRME. There are two challenges with that. First I dont think that scenario is by any means guaranteed because compound growth cuts in favor of $PRME in a second way. $NTLA going up 4x ($1.15Bn to $4.6Bn) requires an additional $3.6Bn in value creation in the markets eyes. $PRME going up 4x ($500M to $2Bn) in the same timeframe only requires an additional $1.5Bn. The market could read that into $PRME merely by starting to believe $NTLA and gene" [X Link](https://x.com/medstudentinvst/status/1957677854134550551) 2025-08-19T05:35Z [--] followers, [---] engagements "It certainly could play out that way. If it does I have a good size stake in $NTLA and would be happy with the returns there. However if it doesnt perhaps bc $PRME starts getting valued off the $VERV buyout price which would get it to 4x from here alone probably whilst nucresiran shows strong results and the market grows increasingly wary of $NTLA I still have a stake in $PRME didnt miss out on the extra 100x / 200x (or more) if $PRME proves successful by getting in at a marginally higher price nor is the $PRME investment predicated on the $NTLA investment if a tail risk event happens and" [X Link](https://x.com/medstudentinvst/status/1957686666052633043) 2025-08-19T06:10Z [--] followers, [---] engagements "I do not think it is true being [--] years ahead makes it hard for $BEAM's valuation to struggle if $PRME has a superior therapy with no bystander edits. There's a lot of historical precedent in biotech that a superior therapy depresses a competitor's valuation in the market's eyes even if it's a couple years behind. This is further true if $WVE proves to also have a superior drug to $BEAM as they are closer than $PRME and would allow a number of patients to bridge to $PRME if they wanted the gene editing solve. Yes nucresiran could only show results in [--] years and $NTLA could have [--] approvals." [X Link](https://x.com/medstudentinvst/status/1957786878238756874) 2025-08-19T12:49Z [--] followers, [---] engagements "August 19th [----]. Exited 50% of $ALNY position for a +94.1% gain. Exited 2/3 of $VKTX position for a +1.9% gain. Remaining 1/3 is down -0.3% after the events of the day. $ALNY - This very much highlighted to me the challenge of investing in the really large biotechs as an individual investor with other professional commitments. I developed significant conviction $ALNY was undervalued based off of the ATTR pipeline and other approved therapies. However $ALNY 's pipeline page has [--] indications. Diligencing the competitive landscape the therapeutic's mechanism of action the market size etc. for" [X Link](https://x.com/medstudentinvst/status/1958051973539721335) 2025-08-20T06:22Z [--] followers, [---] engagements "Jane Street is a quant fund. They are not taking this position because they have a view on the long-term value of the $CRWV. They don't invest based on that. Their algorithms take positions (usually very short-term positions) in companies when signal(s) appear that have a certain probability of being followed by a certain short-term price movement as identified by statistical analysis of massive historical datasets. If you're making this claim from a 13F filing or the like there is a good chance they have already traded out of the position" [X Link](https://x.com/medstudentinvst/status/1958304179216126403) 2025-08-20T23:04Z [--] followers, [---] engagements "August [--] [----] Continued to add to $ABVX and $NKTR. Cost basis moves slightly up for both. As per my post earlier would be a real win if $PRME is able to secure CGD approval. Going to be an important fall / winter for the $NTLA $PCVX and $RARE positions. Very interested to see the data from $PEPG this October. $BEAM and $MGX are my lowest conviction positions at the moment in terms of the long-term competitive advantage / commercial prospects of their pipelines. I am going to continue to hold for now given small position size the demonstrated efficacy of base editing and as a diversification" [X Link](https://x.com/anyuser/status/1958637110669582637) 2025-08-21T21:07Z [----] followers, [----] engagements "42% of companies in the Russel [----] are not profitable. One 25bps rate cut is not going to change that reality nor will it automatically change the reality of mortgage rates debt financing costs etc. So on that view it is strange to see the Russel [----] up 4% today. Now with that said the prices of investable assets move off each market participant's individual buy and sell decisions in response to their individualized moment by sentiment about an asset's forward looking prospects - sentiment defined as the combined effect of reasoned conclusions intuition and emotions So if one rate cut" [X Link](https://x.com/medstudentinvst/status/1958940599618609651) 2025-08-22T17:13Z [--] followers, [---] engagements "Speaking of sentiment shiftthe market participants in which a sentiment shift matters most for biotech investors is the $LLY s $AMGN s $GSK s etc of the world. If their sentiment shifts towards the belief that biotech valuations will start to run significantly it likely accelerates and compresses any M&A activity. Imagine explaining to your board you missed out on getting a highly promising acquisition target on the cheap in one of the worst biotechs markets of all time because you were too slow Seeing significant M&A activity would of course be an additional accelerant to the broader markets" [X Link](https://x.com/medstudentinvst/status/1958971905014116635) 2025-08-22T19:17Z [---] followers, [---] engagements "August 25th [----] $NTLA - nice to see a little bit of a bump due to Friday's insider buy on a day $XBI and $BBC were down 2.5%. $CRSP - Initiated a small $CRSPR position and will continue to add some more over the coming weeks. Target is about [--] to 4% of the current portfolio. This was a tough investment for me to make in a lot of ways. I am not super excited about the commercial prospects of Casgevy (especially with the [--] / [--] rev share with $VRTX) or the nature of the therapy itself - chemo just sucks for patients though recognize the trade off is worth it for some SCD patients. I think in" [X Link](https://x.com/anyuser/status/1960110581081407965) 2025-08-25T22:42Z [----] followers, [----] engagements "August 26th [----] Realized after my post yesterday a larger $CRSP limit order ended up going through earlier in the day. risks of tracking positions on the move / on your phone. So $CRSP position is at 4.5% of portfolio. That is the upper end of the portfolio size I was planning to build to over the next few weeks. So will hold here for now. As said previously the Lp(a) therapy drives my $CRSP investment. Lp(a) data readout in 1H [----] will be key. $CRSP will need to show comparable efficacy to $LLY's siRNA which has had really nice results thus far as it enters Phase III. If $CRSP shows" [X Link](https://x.com/medstudentinvst/status/1960521297722482852) 2025-08-27T01:54Z [---] followers, [---] engagements ".even though a tech contraction would probably drive a rotation of capital back to the biotech sector especially if it occurs alongside commercial validation of innovative platform biotechnologies (e.g. gene editing RNAi etc.) If $NVDA misses earnings today were all gonna die If $NVDA misses earnings today were all gonna die" [X Link](https://x.com/medstudentinvst/status/1960768502102421571) 2025-08-27T18:16Z [--] followers, [---] engagements "August [--] [----] $BBIO - my curiosity is always piqued by the underlying drivers of the between news / event price action with BBIO given the KKR Viking etc. presence. However nice to see it find the $50's and hit a [--] week high. If they are able to compete effectively with $ALNY as data on scripts continues to get released there is likely room to run there $ABVX / $NKTR - the de-risked investments post data release have played out pretty well thus far $NTLA - I am not expecting much until the data releases this fall. However good to see a little resilience in the share price though it remains" [X Link](https://x.com/medstudentinvst/status/1961181414008750207) 2025-08-28T21:37Z [---] followers, [---] engagements "August 29th [----] Tough end to the week for $XBI / $BBC 🤣 $NKTR a lone bright spot. Nice to see $NTLA not get crushed every once in awhile on the down days little more resilient these days on the whole I would say. Though important to remember its the long-term that matters and not the day to day price movements. My hope is that pricing stays around these ranges through the historically tough month of September and into the data readouts that will start coming fast and furious in October. $PEPG likely the earliest readout. Have considered buying some downside protection / trying to play the" [X Link](https://x.com/medstudentinvst/status/1961530037867606134) 2025-08-29T20:42Z [---] followers, [---] engagements "Eh I don't think that's true. $74.7M cash as of 6/30/25. Q2 cash burn of $23.1M so $7.7M per month. $18.2M of Q2 burn is R&D and personnel which will be initial cost savings targets in a wind-down. Make or break data comes early Oct' [--]. If $PEPG continues to burn at $7.7M through 11/30/25 that's $38.5M of burn and $36.2M of cash remaining which is just under their current market cap of $38.7M. Critically half of R&D and an unquantified percentage of personnel spend in Q2 was for DMD therapy discontinued on 5/28. So that's already being wound up $7.7M monthly burn is therefore likely already" [X Link](https://x.com/medstudentinvst/status/1962662800783761679) 2025-09-01T23:44Z [--] followers, [---] engagements "Objectively true. In the words of the great Parcells you are what your record says you are. .and these CDC bureaucrats have a record that is abysmal. The arrogance and self-importance of these @CDCgov bureaucrats is beyond belief. The reality is that the US would have been better off with no CDC at all during the pandemic. Their performance was a Chernobyl-level catastrophe. Their incompetence delayed the start of widespread https://t.co/Juzfbs7nmL The arrogance and self-importance of these @CDCgov bureaucrats is beyond belief. The reality is that the US would have been better off with no CDC" [X Link](https://x.com/medstudentinvst/status/1962681541152104787) 2025-09-02T00:58Z [--] followers, [--] engagements "Many are starting to finally wake up to what $LLY and $GOOG realized wrt $VERV and $CRISPR realized wrt to its own pipeline which is that gene editing in CVD will have unprecedented impact on patient health and enormous commercial return so long as no safety events emerge. Latent safety events is a risk impossible to rule out at this point but the risk-reward is astounding. Atherosclerotic CVD is the biggest killer of human beings in the world atherosclerosis builds up from very young ages yet is largely preventable with early lowering of ApoB in a manner that mimics well-studied naturally" [X Link](https://x.com/medstudentinvst/status/1962888160624451862) 2025-09-02T14:39Z [---] followers, [---] engagements "So under that view wise business decision for $ARWR to license that out" [X Link](https://x.com/medstudentinvst/status/1962890902369628412) 2025-09-02T14:50Z [---] followers, [--] engagements "Best case scenario for Novartis is a donanemab like minimal benefit" [X Link](https://x.com/medstudentinvst/status/1962891818581831790) 2025-09-02T14:54Z [---] followers, [--] engagements "$PRME can meet all five elements of the FDA's RDEP criteria with its CGD program. "Approval under the process may be based on one adequate and well-controlled study plus robust confirmatory evidence (☑) which may include: Strong mechanistic or biomarker evidence ☑ Evidence from relevant non-clinical models ☑ Clinical pharmacodynamic data ☑ Case reports expanded access data or natural history studies ☑"" [X Link](https://x.com/anyuser/status/1963416555624059032) 2025-09-04T01:39Z [----] followers, [----] engagements "https://www.fda.gov/news-events/press-announcements/fda-advances-rare-disease-drug-development-new-evidence-principles https://www.fda.gov/news-events/press-announcements/fda-advances-rare-disease-drug-development-new-evidence-principles" [X Link](https://x.com/medstudentinvst/status/1963416557045817504) 2025-09-04T01:39Z [---] followers, [---] engagements "September 4th [----] Sold $ARWR for 130.1% gain though a very small part of the portfolio. I believe most of the value of $ARWR is contained within its APOC3 (sHTG FCS especially) and ANGPTL3 pipeline (Dyslipidemia / HoFH). Shame they no longer have the Lp(a) therapy though it looks to be behind $LLY's. The FCS and sHTG directed therapies remain very interesting. I need more time to determine whether current valuations are justified by $ARWR's competitive positioning within the sHTG market. I think the ANGPTL3 therapies are somewhat vulnerable to the one-and-done gene editing therapies but" [X Link](https://x.com/medstudentinvst/status/1963717704470446248) 2025-09-04T21:35Z [---] followers, [---] engagements "This will be my thread for my concerns around the INHBE / ALK7 silencers from $ARWR and $WVE. The primary thesis is as follows: [--]. Insulin resistance is counter-intuitively usually characterized by overly active adipocyte triglyceride lipase and hormone sensitive lipase as insulin no longer has the same inhibitory effect on these enzymes found in adipocytes. This overactive ATGL and HSL leads to a huge excess of free fatty acids (FFAs) released in the plasma. These FFAs cause lipotoxicity in the surrounding cells and amongst many other things drive increasing insulin resistance. [--]. Most obese" [X Link](https://x.com/anyuser/status/1963730496489406730) 2025-09-04T22:26Z [----] followers, [----] engagements "Longer post with some more pathway detail made earlier today: I am concerned about $WVE's INHBE silencer's lipolysis enhancing effects in patients that are already diabetic or obese (not born with a LoF variant from birth) as INHBE silencing's mechanisms of action appear to work in opposition to the lipolysis inhibiting effect of a drug class (PPAR- agonists) shown in humans to be highly effective for improvements in blood glucose and insulin sensitivity in diabetic and insulin-insensitive obese patients. The INHBE pathway has a primary effect of inhibiting lipolysis and is governed by the" [X Link](https://x.com/anyuser/status/1963731415708684668) 2025-09-04T22:30Z [----] followers, [---] engagements "Both $ARWR and $WVE are running trials of their INHBE / ALK7 silencers as a monotherapy in obese patients (see attached photos) and so weight loss using INHBE / ALK7 silencers would appear to remain a key objective for the companies. I also think Bruce Givens left $ARWR in [----]. I think the use of NHBE / ALK7 silencers in formerly insulin resistant / obese patients who have restored metabolic health has a reasonable chance of mirroring the metabolically beneficial effects of the LoF INHBE / ALK7 genetic variants by promoting the maintenance and resilience of a healthy metabolic state with are" [X Link](https://x.com/medstudentinvst/status/1964079687841300671) 2025-09-05T21:34Z [---] followers, [----] engagements "September 5th [----] Fully exited the $ALNY position for a gain of 93.4% today though this was a small position in the portfolio. Nice jump to the portfolio in general today. Though the truth is if I am fundamentally wrong on the therapeutic outcomes / commercial viability of a lot of my biggest positions an uplift in the broader biotech market as was seen today won't really matter. I continued to add to $NKTR and $RNA today which brings the cost basis for both slightly up. I will likely continue to add to both so am honestly hoping for a bit of a dip here. I continue to think the risk-reward" [X Link](https://x.com/medstudentinvst/status/1964082840813130204) 2025-09-05T21:46Z [---] followers, [---] engagements "Yeah this is objectively true. Inner city thugs are fake tough. The coward that murdered Iryna Zarutska in cold blood is an obvious example. Went after a defenseless girl on a bus with a knife They would never go after say a jacked combat veteranespecially if the combat veteran is armed to an equivalent degree. Why Because a) these thugs really are soft to their core and b) they know they would get their heads canoed In the rare event they pull this shit without realizing a red-blooded American male is present its always these losers who die. Just like the one Daniel Penny (certified legend)" [X Link](https://x.com/medstudentinvst/status/1965386808725770339) 2025-09-09T12:08Z [---] followers, [---] engagements "This is rending. Penetratingly horrific. The never ending release of violent criminals MUST NOT BE ALLOWED TO CONTINUE. Every single person in America must buy into the following principle: if you choose to engage in an act violence against an innocent in America you get viciously taken down and put away without apology. This coward was released [--] times. 🚨BREAKING: New footage has surfaced of Iryna Zarutska in the moments after career criminal DeCarlos Brown Jr. stabbed her in the neck. Absolutely infuriating and heartbreaking. This is who they let free [--] times. https://t.co/QziLkGouc3" [X Link](https://x.com/medstudentinvst/status/1965501866479157618) 2025-09-09T19:45Z [---] followers, [---] engagements "@capital_usher A good comp if $PRME is successful is $VRTX. They reached a $100Bn+ market cap on the basis of non-curative CF therapies" [X Link](https://x.com/medstudentinvst/status/1965831535883596291) 2025-09-10T17:35Z [---] followers, [---] engagements "They will need to raise (or find other non-dilutive financing like partnerships though would need major partnerships across multiple indications so unlikely to fully fund CF development) in order to bring CF to market. The hope would be that Wilson's and AATD ultimately provide revenue and bolster valuation in the meantime so that the raises are less dilutive" [X Link](https://x.com/medstudentinvst/status/1965832685328482567) 2025-09-10T17:40Z [---] followers, [--] engagements "@DrJessTaylor Thank you for sharing this message. It is so critical that people of all political viewpoints share the fundamental commitment to the right to the freedom of speech and to the sanctity of innocent human life as you clearly do. Your decency is deeply appreciated" [X Link](https://x.com/anyuser/status/1965951700763885975) 2025-09-11T01:33Z [----] followers, [----] engagements "Let me get this straight. You think someone who advocates for pardoning an individual convicted of a violent offense deserves to be murdered for stating that view Im not going to waste time debating Jan. 6th with you. The evilness of your line of thinking is clear even if Jan. 6th happened the way you suggest" [X Link](https://x.com/medstudentinvst/status/1966204693115232328) 2025-09-11T18:18Z [---] followers, [----] engagements "@Stmkrs Check out a post I made earlier today further down this thread on $ARWR s DIO mouse model. Put a few thoughts about that there" [X Link](https://x.com/medstudentinvst/status/1966210600465301994) 2025-09-11T18:41Z [---] followers, [---] engagements "You are so emblematic of the banality of evil. How is it that the most mediocre and ineffectual of people people that would never stride into danger or demonstrate courage amidst great sacrifice are the ones that celebrate horrible violence the loudest. If you have to lie about Charlies points you dont have a point. Also even if he was someone who made the points you claim words are not violence no matter how much you may desperately wish to remain arrogantly removed from dissenting views. Viciously slaughtering someone speaking peacefully whilst his wife and children are watching is real" [X Link](https://x.com/anyuser/status/1966465412872204498) 2025-09-12T11:34Z [----] followers, [----] engagements "It is good to see that you subtly acknowledge his point was not at all that innocents should be murdered or that the fact they are murdered isnt horribly tragic even if you dont have the honor or stones to come right out and say it or answer the question posed to you. It seems you are now saying a public figures phrasing of a point could have been better though you recognize the validity of his underlying point. I think his phrasing was just fine and he actually had the guts to speak to a brutal trade-off unlike you who is too cowardly to actually take a stance on the question posed to you." [X Link](https://x.com/medstudentinvst/status/1966480242807095299) 2025-09-12T12:33Z [---] followers, [--] engagements "The simple truth is that Charlie Kirk represented the views of around 50% of America on most issues and on many issues (like men in women's sport) far more. Yet huge swaths of the left have declared that if you dare to have these views or most any view different than the leftist orthodoxy you either deserve to die or are not worthy of being mourned. You and your views will also be twisted misrepresented and lied about in a desperate effort to justify explicitly or implicitly your murder. Anyone who has spent any time in academia or around leftists knows how real and widespread this sentiment" [X Link](https://x.com/medstudentinvst/status/1966824591663214595) 2025-09-13T11:21Z [---] followers, [--] engagements "I have not wanted to post about amateur biotech investing since Charlie's murder. However to maintain public accountability of performance for whenever I do go back to doing the daily performance update here are the three days I've missed:" [X Link](https://x.com/medstudentinvst/status/1967229751673901143) 2025-09-14T14:11Z [---] followers, [--] engagements "Why Being Supportive of Employers Firing Individuals Celebrating the Murder of Charlie Kirk is Perfectly Consistent with a Belief in Free Speech It is perfectly consistent to be a staunch advocate for the right to free expression of ideas without government-mandated punishment and also believe that employers have a right to fire employees who express beliefs the employer reasonably believes have a high likelihood of materially harming the employees ability to do the job they were hired to do effectively. This is especially true in professions with the responsibility of providing life-saving" [X Link](https://x.com/medstudentinvst/status/1967934772635664445) 2025-09-16T12:53Z [---] followers, [--] engagements "The sheer number of major voices in the conservative movement immediately and correctly calling out the fact that so-called hate speech is protected by the First Amendment whilst speech that actually threatens plans or intentionally incites violence is not demonstrates how sincerely committed most major figures in the American conservative movement are to free speech. This is a good thing and shows the movement is capable of principled self-governance. Hate speech is not prosecutable in America (which is good). Pam Bondi knows this. I am guessing given the statements by Stephen Miller" [X Link](https://x.com/medstudentinvst/status/1967949923346739650) 2025-09-16T13:53Z [---] followers, [--] engagements "You have enemies Good. That means youve stood up for something sometime in your life - Winston Churchill This quote should always come to mind whenever one see the lies and hate directed towards Charlie. For he didnt just stand up for something. He stood up for the most important things - his faith his family his country and his free thought and speech. Always and relentlessly" [X Link](https://x.com/medstudentinvst/status/1968122072166510930) 2025-09-17T01:17Z [---] followers, [--] engagements "Wednesday September 17th Significant reversal in prices post rate cut announcement. The portfolio got as high as 35% return before coming back down. As always $PRME has heavy influence. My suspicion is it will be challenging for $PRME to move far above $5 in the next couple of months unless an announcement is made about accelerated approval pathways in the context of CGD or its other. However I believe its true present value is significantly higher than its current pricing range so a broader market alignment with that view could always occur and therein drive pricing higher. $NKTR continues" [X Link](https://x.com/medstudentinvst/status/1968437689880817920) 2025-09-17T22:11Z [---] followers, [---] engagements "Candace Owens grift is impressively annoying" [X Link](https://x.com/medstudentinvst/status/1968483155368685992) 2025-09-18T01:12Z [---] followers, [--] engagements "This graph is misleading and much of the analysis here is either inaccurate or incomplete. First the y-axis is showing total reported cases of acute Hep B cases in the US not infant Hep-B cases. I understand you likely misread the title of the graph but it should have immediately struck you there were never ever [-----] reported infant Hep B cases in the US. The number of annual infant / perinatal cases before the first Hep B vaccine was developed in [----] and when Hep B was much more endemic was far far lower. That number really matters when you are talking about "catastrophic consequences"." [X Link](https://x.com/medstudentinvst/status/1969209449173615036) 2025-09-20T01:18Z [---] followers, [---] engagements "September 19th [----] $PRME continues to struggle to durably break out past the $5 range. As mentioned previously I think this has a decent chance of remaining true through the '26 INDs unless something happens with CGD. Though as also mentioned previously I think its true present value is a higher than what it currently is if one uses the $VERV buyout as a comp. $NTLA $PEPG and $NKTR all came back down following the run yesterday" [X Link](https://x.com/medstudentinvst/status/1969211852086239682) 2025-09-20T01:27Z [---] followers, [---] engagements "September 22nd [----] New high for the portfolio. Though still a lot of both risk and upside through the end of the year with particularly key data readouts for $PEPG $NTLA $RARE $NKTR $BBIO and $RNA beginning in the next week or so. $NTLA continues to push upwards $PEPG continues to operate at the upper end of the range it has been finding and remains my highest conviction position for greatest returns over the next few weeks" [X Link](https://x.com/medstudentinvst/status/1970237494470885823) 2025-09-22T21:23Z [---] followers, [---] engagements "September 23rd [----] Daily Update New ATH. Though as will be the case for awhile significant risk remains Big finish today for $PRME. I am curious if any of the price action was driven by shifting expectations around CGD based on the recent article and / or back channel chatter around accelerated approval. Time will tell whether its signal or noise" [X Link](https://x.com/medstudentinvst/status/1970580670859624474) 2025-09-23T20:06Z [---] followers, [---] engagements "Cue the shooters motives are unclear media cacophony quickly followed by shooter is MAGA because his third cousin once removed voted for Barry Goldwater FBI saying shooter's rounds had "anti-ICE messages." FBI saying shooter's rounds had "anti-ICE messages."" [X Link](https://x.com/medstudentinvst/status/1970877101671890981) 2025-09-24T15:44Z [---] followers, [---] engagements "@Cernovich Phonak is a good brand if he is going to an independent audiologist / ENT. However take a look at Costco. It's more cost effective and they carry the Jabra Enhance Pro [--] which is a really good option for patients with severe hearing loss. Know a few that had success with it" [X Link](https://x.com/medstudentinvst/status/1970894305851212152) 2025-09-24T16:53Z [---] followers, [----] engagements "@monaco_biotech Competing view: https://x.com/medstudentinvst/status/1970994289413898552 @Hall8Jack @blippd @scamsweeper Comparing dosing for $RNA or $ARWR to dosing for $PEPG has no meaning. $PEPG uses a cell-penetrating peptide conjugated to a phosphorodiamidate morpholino oligomer that sterically blocks the CUG repeats. $RNA and $ARWR use an siRNA that binds to the RISC and degrades the DMPK https://x.com/medstudentinvst/status/1970994289413898552 @Hall8Jack @blippd @scamsweeper Comparing dosing for $RNA or $ARWR to dosing for $PEPG has no meaning. $PEPG uses a cell-penetrating peptide" [X Link](https://x.com/medstudentinvst/status/1970994580326875344) 2025-09-24T23:31Z [---] followers, [---] engagements "Hypomagnesemia was the key potential safety issue observed in DMD51 not eGFR decline. However it was only Grade [--] / [--] hypomagnesemia was transient patients were asymptomatic kidneys showed no signs of structural damage and it resolved without intervention. In regard to eGFR yes you are right in that creatinine went up. However this was not considered reflective of a meaningful safety risk to the kidneys as cystatin C did not go up Cystatin C is a much better indicator of kidney function as creatine fluctuates for all kinds of reasons especially when dealing with peptides and muscle cells as" [X Link](https://x.com/medstudentinvst/status/1971227889178702286) 2025-09-25T14:58Z [---] followers, [---] engagements "September 25th [----] Daily Update New all time high driven by $PEPG result. That is one of the 2H [----] data readouts down. However a lot of readouts to go and significant risk remains. Particularly good to have the $PEPG result on a day when every other position in the portfolio (except for $QURE) was down" [X Link](https://x.com/anyuser/status/1971306190945624561) 2025-09-25T20:09Z [----] followers, [----] engagements "September 26th [----] Daily Update End of week. $PEPG was obviously the highlight. This remains a long-term hold for me" [X Link](https://x.com/anyuser/status/1971666964281257995) 2025-09-26T20:03Z [----] followers, [----] engagements "RA Capital effectively doubled its share count in $PEPG during $PEPG's most recent offering. As I have said elsewhere this company remains high risk given it has only shown Phase [--] data. However it cannot be stressed enough how much upside continues to exist here. DM1 represents at least [-----] patients in the United States with the majority being adult onset. If functional outcomes mirror the unprecedented improvement in splicing $PEPG will almost certainly dominate that market. Given ASO's are usually priced at least a couple hundred thousand dollar years annually that would translate into" [X Link](https://x.com/anyuser/status/1973173962851950818) 2025-09-30T23:51Z [----] followers, 12.1K engagements "$CRSP put out a press release today about its new polymerase based editing technology and claimed the following: "SyNTase editors represent a significant advance over currently described prime editing systems by combining compact Cas9 proteins with a novel class of engineered polymerases. Together these components enable gene editing with greater efficiency and precision specificity while also supporting scalable manufacturing." My current sense this claim of superiority over prime editors is empty marketing fluff and is not supported by existing evidence. $PRME already appears to have" [X Link](https://x.com/anyuser/status/1973476726773830081) 2025-10-01T19:54Z [----] followers, [----] engagements "October 1st [----] Daily Update. Fortunate to have the all-time high run continue. Though as said many times significant risk remains and things could easily reverse if I get a few things wrong this fall or biotech sentiment shifts back towards the negative. Interesting news from $CRSP today. I have posted my preliminary thoughts pending the more detailed data release in October in a separate post. $NTLA with a good gain today. We'll see if it holds. In truth day-to-day price action is rather unimportant relative to the data readouts this fall" [X Link](https://x.com/anyuser/status/1973479747280670949) 2025-10-01T20:06Z [----] followers, [---] engagements "Im not up to speed on the patent law here. Do you know if SyNTase clearly infringes on prime-editing IP I asked Grok and Grok said its unlikely. That was rather shocking to me as its the most obvious way to copycat prime editing.just replace the RNA template and reverse transcriptase with a DNA template and a polymerase" [X Link](https://x.com/medstudentinvst/status/1973485664659194024) 2025-10-01T20:30Z [---] followers, [---] engagements "@tnguy093 Thanks dude. Well see if it holds" [X Link](https://x.com/medstudentinvst/status/1973485899615969480) 2025-10-01T20:31Z [---] followers, [--] engagements "@zhaoweiasu Awesome thanks. Very helpful. I would have been shocked if Prime did not have legal protection against a move like this. Curious if $CRSP will try to claim differentiation nonetheless based on the phrasing of their press release" [X Link](https://x.com/medstudentinvst/status/1973491996389175623) 2025-10-01T20:55Z [---] followers, [---] engagements "Cool thanks. Ill check out those post and will read their DNA polymerase patents in full this weekend. However based on my reading thus far I do think that $CRSP will almost certainly try to claim differentiation from $PRME on the basis of using synthetic nucleotide polymerases. Obviously intellectually and ethically SyNTase is a complete knock-off of $PRME editing. Anyone whos taken Biology [---] would see in a half a second that a DNA polymerase and DNA template will accomplish the exact same thing as an RNA template and reverse transcriptase the first time they read about prime editing." [X Link](https://x.com/medstudentinvst/status/1973551795168305310) 2025-10-02T00:53Z [---] followers, [---] engagements "As per my earlier post on SyNTase I continue to believe that $PRME possesses the leading gene editor technology and $CRSP s SyNTase is a prime editor knock-off that lacks competitive differentiation in specificity has shown no meaningful evidence of competitive differentiation in efficiency and is significantly behind $PRME in development. As always capturing even a few of the thousands of monogenic diseases would drive massive value appreciation for $PRME. However I also think it is likely that $CRSP will aggressively defend SyNTase in court as proprietary and novel in virtue of the fact it" [X Link](https://x.com/anyuser/status/1973567417818222926) 2025-10-02T01:55Z [----] followers, [----] engagements "My man. I assure you I was not attacking you. I think we both agree that SyNTase is an obvious knock-off of prime editors and David Liu / Prime should have the right to this IP from an ethical / intellectual standpoint. I also think we both agree that $PRME retains a dominant lead in prime editing and nothing about SyNTase appears to change that. Im certainly not planning to decrease my position in response to this. Where it seems we differ is I think its highly likely that $CRSP believes they have found a possible legal path around $PRME s patents based on characterizing SyNTase as a" [X Link](https://x.com/medstudentinvst/status/1973577566742065229) 2025-10-02T02:35Z [---] followers, [---] engagements "I do appreciate the post dude. Its great stuff. However I suspect what $CRSP has done is altered the nucleotides in its templates with a few atoms that dont appear as of yet to do a ton functionally (though $CRSP will claim they drive differentiated function) CRISPR is going to subsequently claim that their polymerase engineered to transcribe those templates is a synthetic nucleotide polymerase and therefore outside the purview of $PRMEs DNA / RNA polymerase patents highlighted in your posts and elsewhere and therein $CRSP thinks they have a shot to win a legal fight even though SyNTase is" [X Link](https://x.com/medstudentinvst/status/1973581011024834744) 2025-10-02T02:49Z [---] followers, [---] engagements "I think youre missing my point here dude because Im not really tracking why you keep saying relax. First yes I know it was a press release. A central theme of my posts has been SyNTases differentiated capabilities are very likely overstated in the press release. It was the entire point of the very first post I made about SyNTase. In fact I think the overstatement of capabilities is likely important to $CRSPs legal argument. Second it seems you may be confusing my analysis of CRISPRs likely legal strategy based on the language they used in their press release with some kind of reactionary and" [X Link](https://x.com/medstudentinvst/status/1973592757991043373) 2025-10-02T03:35Z [---] followers, [---] engagements "This discussion is getting repetitive. However as said previously my point is that CRISPR almost certainly believes there is a legal defense that might work here as evidenced by the language of its press release and the fact that it would be grossly incompetent to pursue this without counsel providing a view on legal defensibility. Are you really disagreeing with this If $PRME ends up having to sue to enforce its patent that will cause a headache for them even if they prove victorious. Are you really disagreeing with that" [X Link](https://x.com/medstudentinvst/status/1973596659801051245) 2025-10-02T03:51Z [---] followers, [---] engagements "Hahahahaha okay Wei. Ive been patient with you. Now its clear youre an insecure asshole a mid wit and a waste of my time. Kindly fuck off. Im talking about CRISPR today because its relevant today. Are you slow And yes I know all about Tessera Tome (yes I know theyre winding down) Arbor Metagenomi and the rest. Knowing that is really not a badge of intellectual capability I promise. You can keep fantasizing I am saying $PRME is screwed (lol) so you have something to push back on. That doesnt change the reality that I have consistently stated I believe Prime remains the leading gene editor" [X Link](https://x.com/medstudentinvst/status/1973608409304838570) 2025-10-02T04:38Z [---] followers, [---] engagements "This is a phenomenal article. Kudos to the author. It also shows exactly why $CRSP will almost certainly aggressively make the argument I outlined yesterday to defend SyNTase from legal challenge and why they will lean heavily on their so called proprietary AI-guided structural modeling and large scale screening methods and their use of a synthetic nucleotide template to support the claim that synthetic nucleotide polymerases are novel and represent a significant advance as they interact with non-technical judges / juries. The Amgen case this author describes and Amgens failure to patent" [X Link](https://x.com/anyuser/status/1973751397955862947) 2025-10-02T14:06Z [----] followers, [----] engagements "October 2nd [----] Daily Update Well needless to say $PRME and $NTLAand $CRSP had a day. $PEPG a nice rise as well and $PCVX continues to show some strength I do feel the need to always caveat that this continues to be a high risk portfolio and things could turn quickly. However pleased with the results thus far" [X Link](https://x.com/anyuser/status/1973842631013593431) 2025-10-02T20:08Z [----] followers, [----] engagements "It looks like Viking also increased its position in $PEPG from [-------] at end of Q2 [----] to [-------] shares. Filed a 13G today" [X Link](https://x.com/anyuser/status/1974245717897044153) 2025-10-03T22:50Z [----] followers, [----] engagements "@hannibalspeaks Yes exactly. I am George Washington's only living heir" [X Link](https://x.com/medstudentinvst/status/1974248990481752465) 2025-10-03T23:03Z [---] followers, [--] engagements "October 6th [----] Daily Update Reposting as the $ABVX close price was not updated" [X Link](https://x.com/medstudentinvst/status/1975299056487571602) 2025-10-06T20:36Z [---] followers, [---] engagements "@GeneInvesting Give it a rest Tony. This brand of insecure makes me want to dump every share of $NTLA I own. The stock could go to $200 and your returns still wouldn't come close to his" [X Link](https://x.com/anyuser/status/1975670758807638418) 2025-10-07T21:13Z [----] followers, [----] engagements "Today is a good example of the benefits of the approach I outlined last night. I have effectively no insight into what non-event driven pricing in a biotech stock will do day to day. I could just as easily have seen $PRME and $NTLA being down 12% today rather than up 12% and 28% (for now) ______ As a small aside I do have a theory that biotech stocks especially over the last few years are an ideal sector for quant funds because of (a) the volatility and (b) there are likely extended timeframes where the drivers of intraday pricing are far more statistically predictable given the profile of" [X Link](https://x.com/anyuser/status/1975989705381425529) 2025-10-08T18:20Z [----] followers, [---] engagements "Closed out the $RNA position today for effectively no gain given I think there are higher return opportunities in biotech. Put half into $QURE. The remaining half is in cash and will look to deploy in the future" [X Link](https://x.com/anyuser/status/1975991061450571867) 2025-10-08T18:25Z [----] followers, [---] engagements "October 8th [----] Daily Update All-time high. $NTLA and $PRME with big days. Though I would not be surprised to see continued volatility and a retraction. The gains for $NTLA are really little more than just noise until the data readouts in a few weeks. As mentioned in a previous post I exited $RNA at an effectively flat valuation. This is largely a function of believing there are biotech companies with similar risk profiles that have a higher upside rather than a negative view of $RNA. Certainly could be bought out and there is likely upside there. I initially entered $RNA as a relatively" [X Link](https://x.com/medstudentinvst/status/1976018897498255644) 2025-10-08T20:16Z [---] followers, [---] engagements "$ABVX didn't update again and just realized so here is the corrected daily update for $ABVX's actual close price. No change to headline total return" [X Link](https://x.com/medstudentinvst/status/1976020094007275595) 2025-10-08T20:21Z [---] followers, [---] engagements "October 9th [----] Daily Update All-Time High. Still super risky portfolio given it's biotech. $NKTR and $NTLA probably the companies with the biggest potential catalysts over the next few weeks" [X Link](https://x.com/anyuser/status/1976386018065707137) 2025-10-09T20:35Z [----] followers, [----] engagements "Mostly just wanted to put the cash towards $NKTR. My view on $VKTX continue to be it likely has a market and upside if it maintains headline efficacy lead in the GLP-1 / GIP agonist class regardless of any side-effect incidence matched comparisons though competition from big players is intense and CMC remains important. I think it's likely continue to show in upcoming data readouts" [X Link](https://x.com/medstudentinvst/status/1978474237909176407) 2025-10-15T14:53Z [---] followers, [---] engagements "@AdamBLiv Alternatively you could just buy an asset that has a 127% CAGR. Neither a 401k nor bitcoin necessary. I am the quant now" [X Link](https://x.com/medstudentinvst/status/1978524731780345899) 2025-10-15T18:13Z [---] followers, [--] engagements "Alternatively you could just buy an asset that has a 127% CAGR. Neither a 401k nor bitcoin necessary. I am the quant now. People dont like hearing this but putting money your 401(k) just to get the employer match is stupid. Most people dont realize their 401(k) match is just a leash. Free money sounds nice until you realize its denominated in melting ice cubes and you cant touch it for [--] People dont like hearing this but putting money your 401(k) just to get the employer match is stupid. Most people dont realize their 401(k) match is just a leash. Free money sounds nice until you realize its" [X Link](https://x.com/medstudentinvst/status/1978524780434255958) 2025-10-15T18:14Z [---] followers, [---] engagements "I do think given the $VERV $1Bn buyout the investment is derisked somewhat regardless of the day to day price movement in a fire sale scenario unless there's a major prime editing platform safety event It's interesting if you go back you'll see $ALNY had its issues and its volatility for a long time. So a sign of hope there https://twitter.com/i/web/status/1983989106851115029 https://twitter.com/i/web/status/1983989106851115029" [X Link](https://x.com/medstudentinvst/status/1983989106851115029) 2025-10-30T20:07Z [----] followers, [---] engagements "Come on man. Thats not good faith. You really believe what I wrote means Im comping $PEPG today to $ALNY or $BBIO Might as well say Im comping $PEPG to $PFE while youre at it. It should be abundantly clear I am drawing the comparison between $PEPG s EDODM1 and $BBIO and $ALNY s ATTR-CM drugs to assess the ability of an approved post-phase III best-in-class drug in DM1 if EDODM1 makes it that far to gain 10% market share in the US market which was the point you questioned. On the prevalence point using that worldwide number is purposefully obfuscatory when I was citing US numbers for DM1. We" [X Link](https://x.com/medstudentinvst/status/2007641078246035514) 2026-01-04T02:31Z [----] followers, [---] engagements "The content of the article itself is fine. Just says $ABVX explicitly declined to comment on any M&A / auction processes and that Marc confirmed a formal review by the French government can't occur until there is a publicly announced offer which speaks directly to the statement by the French government that there had been no discussions with $LLY. It's the headline Reuters chose that's the issue as it erroneously suggests Marc denied the existence of any auction process / M&A discussions on the basis of Marc's comment that $ABVX can't worry about market "noise" and they just need to execute" [X Link](https://x.com/medstudentinvst/status/2013673658539999524) 2026-01-20T18:03Z [----] followers, [---] engagements "$PEPG position initiated this morning based on what I see as great reward to risk ratio leading into critical October [----] data readout. If drug demonstrates efficacy think at least 4x return possible off that data readout alone. Long term return if successful much greater. Key is to keep position size small enough for portfolio to absorb losses if fails while still having enough size to capture upside at the portfolio level. If drug fails downside perhaps protected by negative EV and non-zero likelihood RA Capital / Viking Global / Point72 successfully push $PEPG to return cash to" [X Link](https://x.com/anyuser/status/1956036808745345550) 2025-08-14T16:54Z [----] followers, 16.8K engagements "$PCVX Believe it is likely the NIH statement referenced below that has driven the slight uplift seen over the past week. Below can see Prevnar ( $PCVX 's key competitor) numbers revenue numbers. $PCVX market cap is currently $4.25Bn. Taking 30% of this market share would yield $1.9Bn in revenue and $9.5Bn EV off 5x revenue multiple. 50% of this market share would yield $3.2Bn in revenue and $16.0Bn EV off 5x revenue multiple. 75% would yield $4.0Bn revenue and $20Bn EV off 5x revenue multiple. As stated previously historical norm for commercial stage biotechs is 5.5x - 7.5x revenue multiple." [X Link](https://x.com/medstudentinvst/status/1956571009126945196) 2025-08-16T04:17Z [---] followers, [---] engagements "August 20th [----] Used proceeds from selling half of Alnylam position (+94.1%) yesterday into $NKTR and $ABVX (about half into each). $VKTK thoughts from yesterday - This trade really emphasized to me the importance of staying tight on three things: [--]. Entry price - A good business is a bad investment if it is bought at too high a price. A mediocre business can be a good investment if bought at a cheap enough price. Of course one can take that principle too far overly optimize for entry price and miss out on an opportunity because the stock runs. In such cases if the expected return is" [X Link](https://x.com/medstudentinvst/status/1958276267393929538) 2025-08-20T21:13Z [---] followers, [----] engagements "August 22nd [----] $PRME getting a nice little rip at the end of the week thanks to Jerome. $WVE has had a nice run recently. The limited reaction from $PCVX $NTLA and $RARE (fall / winter readout stocks) raises the probability of muted upside until their fall readouts hit. $ABVX continuing to inch up" [X Link](https://x.com/anyuser/status/1958999216401129589) 2025-08-22T21:06Z [----] followers, [----] engagements "Another thought from the drafts: There are issues with mice data provided by $ARWR. First competing mice data appears to show the lipolysis driven by INHBE / ALK7 silencing ( which $WVE / $ARWR would acknowledge is the purpose of the therapy) does lead to lipotoxicity and signs of insulin resistance like hepatic steatosis within certain metabolic contexts. I've put one of the studies (there are multiple) showing this below. Additionally take a look at the attached screenshot of $ARWR 's glucose / insulin sensitivity data. The tests they are running are probably not sensitive enough to detect" [X Link](https://x.com/medstudentinvst/status/1966173965220720984) 2025-09-11T16:16Z [---] followers, [----] engagements "$QURE news is good news for patients more bad news for $WVE $WVE: [--]. RNA editing platform has demonstrated inferior efficacy relative to DNA editing / base editing thus far [--]. Obesity / INHBE silencer may drive increased insulin resistance in patients who are already obese as laid out in previous posts. Competition with $ARWR at baseline [--]. One and done Huntington Disease therapy from $QURE sets a high bar for efficacy $WVE will need to match or exceed $QURE is one and done vs $WVE s continuous dosing regimen and $QURE is in more advanced stages than $WVE [--]. DMD therapy (exon 53) only" [X Link](https://x.com/medstudentinvst/status/1970854564816404875) 2025-09-24T14:15Z [---] followers, [---] engagements "$PEPG Well there you have it. It shall indeed likely be the highest gaining position over this short-term time frame. It was just such an unbelievable opportunity to be able to buy it in the low $1's. For reference $PEPG's key competitors (which it appears to blow out of the water) are multi-billion dollar companies ( $RNA and $DYNE). https://twitter.com/i/web/status/1970962013049573862 https://twitter.com/i/web/status/1970962013049573862" [X Link](https://x.com/anyuser/status/1970962013049573862) 2025-09-24T21:22Z [----] followers, 12.6K engagements "September 30th [----] Daily Update This can't continue forever. but another all-time high today largely on the strength of $PRME. Good to see $PCVX back in the green too after announcing a $1Bn manufacturing deal with Thermo Fisher. My sense is $PCVX will be a beneficiary of a couple things: [--]. The market's realization that NIH / FDA isn't anti-vaccine it's anti-stupid vaccine. Or said more academically it's anti-vaccine for vaccines that have an unclear risk-reward tradeoff. In the United States pneumococcal pneumonia is estimated to result in approximately [------] hospitalizations each year" [X Link](https://x.com/medstudentinvst/status/1973174061921357930) 2025-09-30T23:52Z [----] followers, [----] engagements "Id say $QURE $PCVX $RARE $NKTR $ABVX are high growth potential with somewhat more limited risk than the others. However biotech is always a very very high risk sector and I cant predict any outcome in biotech with certainty so if youre going invest in those please only do so if youre fully comfortable taking big losses in the positions. Obviously none of this is financial advice. If the risk parameters were widened just a bit I would also throw $NTLA in there as good risk / reward. Quite high upside and key data readouts upcoming in the next few weeks with good visibility into what the" [X Link](https://x.com/anyuser/status/1974269818946703719) 2025-10-04T00:26Z [----] followers, [----] engagements "Hypothesis for a contributor to $PEPG s differentiated splicing improvement in DM1. $RNA and $DYN use DMPK1 RNA silencers which degrade and destroy the DMPK mRNA. In contrast $PEPG s ASO binds to the CUG repeats and sterically blocks the hairpin loop formation allowing the DMPK mRNA transcript to be translated normally. Most biological systems have negative feedback loops. So an increase in expression production or activity or a molecule will trigger inhibition of the expression production or activity of that molecule. Given $PEPG allows the DMPK mRNA to operate normally and produce the DMPK" [X Link](https://x.com/anyuser/status/1974397895387292146) 2025-10-04T08:55Z [----] followers, [----] engagements "They have a very different risk / reward profile. $MREO does not have an existing commercial business generating hundreds of millions of dollars. If setrusumab fails $MREO's stock price is therefore likely to fall much more than $RARE's. We saw that after setrusumab missed at IA2. Alvelestat almost certainly will be non-competitive with $BEAM $PRIME 's and $WVE 's AATD therapies" [X Link](https://x.com/medstudentinvst/status/1974910826550075597) 2025-10-05T18:53Z [---] followers, [---] engagements "The failure of $SKYE s therapy is notable today in the context of INHBE / ALK7. The primary lipolysis enhancing effect of INHBE / ALK7 silencers appears to be driven by an increase in intracellular cAMP and PKA via inhibition of pathways that decrease cAMP and PKA concentrations. As noted elsewhere PKA drives activation of lipolysis enzymes (e.g. HSL and ATGL via perilipin). $SKYE s therapys primary downstream effect is also enhanced lipolysis through upregulation of cAMP and PKA concentrations via inhibition of pathways that decrease cAMP / PKA concentrations. It just works the CB1 receptor" [X Link](https://x.com/medstudentinvst/status/1975174023286853681) 2025-10-06T12:19Z [---] followers, [---] engagements "Right. OI represents the predominant driver of $RARE s upside. However the baseline commercial business of $RARE is probably worth around $28 per share. That provides substantial downside protection if setrusumab fails at the Final Analysis. In such a scenario $RARE will probably trade no lower than mid-teens immediately following the news and still has a reasonable chance to recover to those high twenties levels and higher if Angelman proves to be a commercially successful therapy. In contrast If setrusumab fails $MREO will be wiped out and thats particularly true if Alvelestat is considered" [X Link](https://x.com/medstudentinvst/status/1975244521681395728) 2025-10-06T16:59Z [---] followers, [---] engagements "Im not really sure what there is to debate here. Yes setrusumab has a reasonable probability for success hence my investment in $RARE. However it quite obviously does not have a 100% chance of success. Consequently $MREO carries more risk into the FA with $MREO s downside case being a complete decimation of the stock and $RARE s downside case ranging from mid teens to effectively flat. Do you disagree with that As I have said I dont want $MREO s binary risk exposure when $RARE has likely 2x upside with a baseline commercial business generating hundreds of millions of dollars in revenue. If" [X Link](https://x.com/medstudentinvst/status/1975260106842341604) 2025-10-06T18:01Z [---] followers, [---] engagements "Im not really sure what there is to debate here. Yes setrusumab has a reasonable probability for success hence my investment in $RARE. However it quite obviously does not have a 100% chance of success. Consequently $MREO carries more risk into the FA with $MREO s downside case being a complete decimation of the stock and $RARE s downside case ranging from mid teens to effectively flat. Do you disagree with that As I have said I dont want $MREO s binary risk exposure when $RARE has likely 2x upside with a baseline commercial business generating hundreds of millions of dollars in revenue. If" [X Link](https://x.com/medstudentinvst/status/1975260368822808753) 2025-10-06T18:02Z [---] followers, [---] engagements "$ALNY has returned 76.5x since inception. $10000 invested at $6 would be $766500 today. $50000 would be $3.83M today. $100000 would be $7.66M The picture attached is $ALNY 's stock chart. This represents the argument for why to stay long in the face of volatility when invested in early stage biotechnology companies which you believe have a very good chance of seeing enormous value accretion over the long-term based on TAM competitive differentiation current valuation etc. $ALNY 's share price saw significant volatility over the years. It collapsed soared plateaued and everything in between." [X Link](https://x.com/anyuser/status/1975685770737258774) 2025-10-07T22:12Z [----] followers, [----] engagements "October 10th [----] Daily Update I allocated the remaining $RNA cash to $JANX today. If $NKTR continues to move down I will likely exit the tiny $BEAM and $VKTX holdings and put those towards $NKTR. Portfolio as a whole drew back about 7.3% today largely driven by the top [--] positions in the portfolio ( $PRME $NTLA $PEPG). I expect $PRME and $PEPG to continue to experience significant volatility over the short to medium term and consider these price movements irrelevant unless my long-term thesis on the business changes or new data emerges. Though it means the portfolio as a whole is likely to" [X Link](https://x.com/anyuser/status/1976791208090353940) 2025-10-10T23:25Z [----] followers, [----] engagements "Just closed small remaining positions in $VKTX and $BEAM. Proceeds went into $NKTR. Portfolio weights will be updated in daily update tonight" [X Link](https://x.com/medstudentinvst/status/1978472233396732196) 2025-10-15T14:45Z [---] followers, [---] engagements "$PCVX continues to inch-up. They still have data to show of course. However in my personal view it was an absolute steal a steal to be able to get this in the 30's for a company as large it is with such a strong a cash position. Surprised it took as long as it did to see some of the sector gains biotech in general has seen especially after NIH made its vaccine position abundantly clear. $RARE is also inching up. Setrusumab is still an open question and is by no means guaranteed. However from a risk-reward perspective given downside is protected by the commercial business and has two other" [X Link](https://x.com/medstudentinvst/status/1978487036764934419) 2025-10-15T15:44Z [----] followers, [---] engagements "Lorenzo "Botswana" (who has $1Bn of AUM a PhD [--] other acronyms and clips $20M of annual management fees given he obviously is a [--] and [--] guy minimum) shorted $SOC at $30 as announced after the fact. Word is he was also long $SPRB as announced the day after it increased [--] trillion percent. Are there any investor materials available for a highly interested prospective LP such as myself I shorted $SOC at $30 because the dumbest motherfuckers Ive ever seen own that stock. They do not know the method. Elevate. 💯 I shorted $SOC at $30 because the dumbest motherfuckers Ive ever seen own that" [X Link](https://x.com/anyuser/status/1978494913189703975) 2025-10-15T16:15Z [----] followers, [----] engagements "Early Daily Update for posterity as I might never see this again and this whole thing could easily collapse tomorrow. The current portfolio is all green. The big glaring red mark is that dumb throwaway $MGX position I exited. The intrusive thoughts to re-enter and try and ride it until I erase that -13.5% are flying simply fast and furious rn https://twitter.com/i/web/status/1978540347014946998 https://twitter.com/i/web/status/1978540347014946998" [X Link](https://x.com/medstudentinvst/status/1978540347014946998) 2025-10-15T19:15Z [----] followers, [---] engagements "Wednesday October 15th [----] Daily Update All-time high. Though always needs to be said this is a high risk portfolio and there are a number of upcoming events that could turn the ship right around. As mentioned closed the very small $BEAM and $VKTX positions today and put virtually all remaining cash into $NKTR" [X Link](https://x.com/medstudentinvst/status/1978553595974033891) 2025-10-15T20:08Z [---] followers, [----] engagements "October 16th [----] Daily Update Markets go weeeeeeeeeeeeeeee portfolio comes back into the 70s after all but $RARE is red today. $RARE (and $PCVX to an extent) showed the most resilience. Again these are names that I think are likely to have high institutional interest and have not experienced the same run as some of the others. So not entirely surprised to see that" [X Link](https://x.com/medstudentinvst/status/1978918054227775740) 2025-10-16T20:16Z [----] followers, [---] engagements "Totally agree DM1 is a huge market and $pepg very interesting. Re $WVE and INHBE may want to check out the attached post. I think there is a fairly high likelihood that $WVEs approach of further increasing adipocyte / hepatocyte lipolysis through INHBE silencing will worsen insulin sensitivity in patients who are already obese and almost certainly already have excess lipolysis as the baseline excess lipolysis is a major causal factor in insulin resistance / diabetes. I think there is a core difference in molecular profile between patients who have LoF INHBE gene variants and therein" [X Link](https://x.com/anyuser/status/1979306544622895157) 2025-10-17T22:00Z [----] followers, [----] engagements "Sorry I am just seeing this. Thank you I think it has a better chance in a maintenance role once adipocyte lipolysis has normalized somewhat post GLP-1. However a key issue it will need to overcome is that obesity tends to leave a lot of durable molecular signatures that promote weight gain even after an obese patient has lost weight that are not present in the never obese patients who experience the LoF INHBE mutations. Thats the main reason keeping weight off is so hard for the formerly obese. I think durable molecular signatures that have effects on appetite drive play the most biggest" [X Link](https://x.com/medstudentinvst/status/1980680413048651876) 2025-10-21T16:59Z [---] followers, [--] engagements "As I said at the time of the press release it was blindingly obvious $CRSP was going to aggressively argue SyNTase was a substantively different technology than prime editors and outside the scope of $PRME's patents regardless of whether that position is ethical or intellectually honest. As I also said at the time this may prove to be a headache for $PRME even if it wins out or gets licensing fees in the end. Legal outcomes are impossible to predict and legal processes are expensive. $CRSP can bear legal costs a lot more comfortably than $PRME can. $CRSP 's intentions and their likely legal" [X Link](https://x.com/anyuser/status/1981025094693470698) 2025-10-22T15:49Z [----] followers, [----] engagements "For headline clarity (see back half of the post) I believe $PRME remains the clear leader in gene editing. I think it remains a phenomenal venture style investment over the long-term for the [--] reasons highlighted derisked barring a black swan safety event based on the $VERV buyout at the depths of the bear market and remains my largest position. I have not sold a share and nothing about this situation causes me to change that weighting. This is commentary about CRISPRs intentions the legal situation and possible impacts on short to medium term cash and operational dynamics for $PRME which may" [X Link](https://x.com/medstudentinvst/status/1981054782711189809) 2025-10-22T17:47Z [---] followers, [---] engagements "October 22nd [----] Daily Update Those gene editing stocks sure can be volatile. Large pullback the last coupe of days. Total return actually dipped into the high 60s at one point. If $NTLA $RARE and $PCVX can show good data and $NKTR gets some good news in AA one small upside of that will be a degree of smoothing out in the some of the headline volatility somewhat that is an inherent part of owning a very large position in $PRME. Though in truth the volatility doesn't really matter only the end outcome. Very curious to see what $NKTR has to say about the asthma sub-group in its Nov update" [X Link](https://x.com/medstudentinvst/status/1981091117702730131) 2025-10-22T20:11Z [----] followers, [---] engagements "October 23rd [----] Daily Update All eyes on early November👀( $NTLA $NKTR)" [X Link](https://x.com/anyuser/status/1981451510992032079) 2025-10-23T20:03Z [----] followers, [----] engagements "October 24th [----] Daily Update The positions are set and the Q4 data gauntlet begins. The portfolio could look very different by the end of the next two months for better or worse. $BBIO will announce the results of its LGMD2i/r9 therapy on Monday morning. This will have a relatively small impact on the portfolio either way due to the position size and its small relative contribution to $BBIO's valuation. However early November will bring higher impact data readouts. The most notable is $NTLA" [X Link](https://x.com/medstudentinvst/status/1981863278562152757) 2025-10-24T23:20Z [---] followers, [---] engagements "Bad news from $NTLA. This will have significant price impact and those effects will ripple across the gene editing sector. As it stands my position is at a 1.8% loss. So where do we go from here Grade [--] elevations have happened before in Q2 '22 (technically I don't believe it was specified as Grade [--] but I am pretty confident it was) and May [----]. Thus Grade [--] elevations are a known phenomenon and at this point have occurred enough that it appears to be an inherent risk of the therapy. If this Grade [--] elevation is a mere plasma elevation with have no clinical symptoms or structural liver" [X Link](https://x.com/anyuser/status/1982793202475172299) 2025-10-27T12:55Z [----] followers, [----] engagements "I have closed my $NTLA position for a small gain (will be shown in portfolio update tonight). Below I will break-down my rationale for exiting and detail a hypothesis for what may have happened My investment in $NTLA was largely based on the following: [--]. $NTLA demonstrated the ability to stop (not just slow) functional decline over the timeframe studied in an unprecedented percentage of patients with transthyretin amyloidosis a fatal disease that leads to heart failure polyneuropathy and a whole host of downstream consequences not well described in the medical literature or recognized" [X Link](https://x.com/anyuser/status/1982833331323490448) 2025-10-27T15:34Z [----] followers, [----] engagements "If this safety event does not prove to be what it initially seems or there is a clear way to identify patients at risk for the disease I will revisit this decision. However for now I am out" [X Link](https://x.com/medstudentinvst/status/1982833334251347983) 2025-10-27T15:34Z [---] followers, [---] engagements "Finally a hypothesis for what happened. I posted elsewhere (will try to find it today) that one of the under appreciated risks of the gene silencer companies (first-gen CRISPR / $NTLA / $CRSP) is that the way they are intended to work is through the stochastic alteration of a targeted genes genetic sequence through indels which renders it unable to make a functional mRNA transcript and leads to the natural degradation by the cell of that transcript however it is well described that certain mutated mRNA transcripts can lead to toxic effects and its possible the random deletion or insertion of" [X Link](https://x.com/anyuser/status/1982833337593983157) 2025-10-27T15:34Z [----] followers, [---] engagements "It remains to be seen what emerges regarding the underlying cause. I think one hypothesis that has merit (but is simply a hypothesis) is the indels introduced by $NTLA's cas9 at the site of the DSB create a new genetic sequence which codes for a toxic mRNA perhaps limited to a small subset of patients because a second baseline mutation is required for toxicity (the TTR protein has hundreds of natural occurring variants). Mutations causing toxic mRNA is a well-described phenomenon in other contexts If this hypothesis were to be correct it would have no effect on $PRME as prime editors are" [X Link](https://x.com/medstudentinvst/status/1982878484260368829) 2025-10-27T18:34Z [---] followers, [---] engagements "October 27th [----] Daily Update I posted my thoughts on the disappointing $NTLA news my original thesis why that thesis changed post safety event and a hypothesis for what happened mechanistically earlier today. That is a more complete breakdown. However at the portfolio level wrt $NTLA today was a good example of entry price playing an important role in the risk-reward profile of an investment. Despite a potential program threatening safety event was able to close the position with a 2.2% gain and the portfolio as a whole was only down about 5% from Friday. Believe upside was worth such a" [X Link](https://x.com/anyuser/status/1982910876911874052) 2025-10-27T20:42Z [----] followers, [----] engagements "The short answer is I do with the primary reasons being: [--]. Large market size of AD [--]. Unique MoA [--]. Strong efficacy to date [--]. Very favorable side effect profile (personally I would choose ISR risk profile over the others) [--]. Likelihood of t-reg selective IL-2R agonist success in other indications (alopecia areata in the nearer-term and potentially others beyond) One of the elements I find most exciting about t-regs is they are biological entities designed to modulate their effector functions in response to the surrounding immune environment in highly complex ways. Thus while IL-2R agonism" [X Link](https://x.com/medstudentinvst/status/1982919581694890408) 2025-10-27T21:17Z [---] followers, [---] engagements "The first liver enzyme elevation was in of itself irrelevant nex-zs superior efficacy to existing alternatives as it was transient and had no clinical consequence. If this event fit the May [----] event profile I would not have sold a share of my position. Vutri does not do the same job as nex -z and this is well established in the clinical data. This should not be in question and if it is it just means you are neither familiar with the data nor the disease. Nex-z stabilizes functional decline for a significant percentage of patients. Vutrisiran does not. I assure you if a family member was" [X Link](https://x.com/medstudentinvst/status/1982924474799235266) 2025-10-27T21:36Z [---] followers, [--] engagements "I think $NTLA bears and bulls are talking past each other right now and many on both sides are missing a key piece of the equation - nucresiran. Bulls say $NTLA's nex-z results in a significantly lower percentage of patients showing disease progression relative to $BBIO's acoramidis and $ALNY 's vutrisiran. This is empirically true and it really does matter. Progression of heart-failure is tremendously serious and it is critically important to patient outcomes to fully arrest it as early as possible to prevent the panoply of downstream consequences like cognitive decline kidney dysfunction" [X Link](https://x.com/anyuser/status/1983124850286694751) 2025-10-28T10:53Z [----] followers, [----] engagements "October 28th [----] Daily Update Position in $RCKT initiated today. Very high-risk. Quick and dirty thesis posted earlier today. Three shots on goal to see returns. Position size key so failure across all three will not materially impact portfolio to the downside but success in at least one will. $BBIO announcing data on ADH1 tomorrow. Very nice suite of therapies at this point. $QURE continues to move in a positive direction $PEPG back down. Honestly I had a tough time seeing that jump post $RNA acquisition being durable. It seems to me much too abstract in its relevance to $PEPG to support a" [X Link](https://x.com/medstudentinvst/status/1983276738647134255) 2025-10-28T20:56Z [----] followers, [---] engagements "Whoops typo in the first sentence - PKP2-ACM as seen below. Quick and dirty for real" [X Link](https://x.com/medstudentinvst/status/1983277266428932114) 2025-10-28T20:58Z [---] followers, [---] engagements "I certainly thought so before this safety event. If the only notable side effect was rare and transient liver enzyme elevations that resolved without medical intervention i think there was a good probability that nex-z would have found success and seen large returns relative to its recent lows. I personally would have had a tough time prescribing tafa / acora / vutri over it given nex-z gives a patient a meaningfully better chance at seeing no disease / heart failure progression (as measured by pro-ntBNP / functional tests) at least over the timeframe studied and that really matters" [X Link](https://x.com/medstudentinvst/status/1983349589311692873) 2025-10-29T01:46Z [---] followers, [--] engagements "Really fantastic results so far this week from $BBIO through and through. A very well-run company that seems to do things the right way. Really appreciated the way they provided a free lifetime supply of acoramidis to patients. Makes them easy to root for. I love the simplicity of encaleret. It's an oral drug that directly targets the key underlying pathophysiology of ADH1 and its MoA means it's unlikely to significantly perturb endogenous pathways whilst doing so. My eyes now turn to what is going to happen with tafamidis' patent protection. If Pfizer is able maintain tadamidis patent" [X Link](https://x.com/medstudentinvst/status/1983506996545356201) 2025-10-29T12:11Z [---] followers, [---] engagements "October 29th [----] Daily Update Pullback continues in the portfolio though feel pretty good about the positions as they stand. $PRME continuing to trend down post $NTLA news is the biggest contributor to the ongoing pullback. For a whole host of reasons I posted about yesterday I do not believe there is a read through from $NTLA to $PRME. One of the reasons I listed and haven't seen discussed much (most of the focus is on the LNP / the cas9 mRNA / protein immunogenicity) is it is possible $NTLA's issues flow from cas9 induced indels which are a design feature of first-gen CRISPR and not prime" [X Link](https://x.com/medstudentinvst/status/1983647880763019279) 2025-10-29T21:31Z [---] followers, [----] engagements "Just FYI $ALNY 's nucresiran (only 1-2 years behind nex-z) will almost certainly match nex-z's efficacy based on depth and speed of TTR knockdown. I would have a very hard time feeling comfortable prescribing nex-z if I had an equally effective drug that didn't carry the risk of a DILI in nucresiran (2x per year dosing). $NTLA's valuation depends on long-term nex-z prescriptions. Something to consider as I think $NTLA is at risk in a way it has never been before" [X Link](https://x.com/medstudentinvst/status/1983663166379725179) 2025-10-29T22:32Z [---] followers, [---] engagements "An additional point in support is $ALNY 's nucresiran will almost certainly show the same efficacy as nex-z based on rapidity and depth of TTR knockdown with no DILIs. So nex-z's point of differentiation in having a clinically meaningful efficacy advantage over acora / tafa / $ALNY's vutri gets obviated as soon as nucresiran comes to market 1-2 years behind and there then isn't even an efficacy argument to support a prescription of nex-z" [X Link](https://x.com/medstudentinvst/status/1983943857453609462) 2025-10-30T17:07Z [---] followers, [---] engagements "I know man. it's tough to see. I expect that this will be a bumpy ride for awhile. Deserved or not I unfortunately think $NTLA was acting as a bellwether / canary in the coal mine of sorts for in vivo gene editing in the markets' eyes and I expect $NTLA's very real issues with DILI to ripple through the sentiment around the rest of the sector for awhile. $PRME will no longer get the market confidence boost from an in vivo editor making in to market without notable issues. I continue to believe there is a good chance that the DILI is a function of the stochastic indels first-gen CRISPR is" [X Link](https://x.com/medstudentinvst/status/1983985204906631536) 2025-10-30T19:51Z [---] followers, [---] engagements "Also just added to $ABVX. Basically all time highs but perhaps the waters are swirling and want to favor being in market with capital during a bull run rather than out" [X Link](https://x.com/anyuser/status/1983986163498283387) 2025-10-30T19:55Z [----] followers, [----] engagements "October 30th [----] Daily Update Explained reasoning behind changes made today in previous posts. [--]. Closed $CRSP [--]. Opened $PASG [--]. Opened another $ABVX position" [X Link](https://x.com/anyuser/status/1983994713037476219) 2025-10-30T20:29Z [----] followers, [----] engagements "The Lilly trial is still enrolling unless youve seen something Ive missed The death in their trial was not considered related to the therapy and the DRG issues have not appeared in the Dose [--] (high dose cohort w/ supraphysiplogic progranulin expression) for $PASG. Dose [--] is actually a 50% lower dose" [X Link](https://x.com/medstudentinvst/status/1984308661812949063) 2025-10-31T17:17Z [----] followers, [---] engagements "Reposting an excerpt of a hypothesis I put out a few days ago regarding the underlying cause of $NTLA s DILI. I could have sworn I also posted about this specific risk some months back though have not been able to find the post and it may have just been in my notes / thesis amidst possible risks. The hypothesis didnt drive returns as until the DILI it was theoretical to my knowledge. However it prevented me from losing money on the investment as that hypothesis along with the existence of nucresiran drove my $NTLA exit post DILI. It also shows why there is a low probability of a read through" [X Link](https://x.com/medstudentinvst/status/1984408295512150418) 2025-10-31T23:53Z [---] followers, [---] engagements "October 31st [----] Daily Update Added a little more to $PASG to close out the week" [X Link](https://x.com/medstudentinvst/status/1984431346559709464) 2025-11-01T01:24Z [----] followers, [---] engagements "@plainyogurt21 @aditharun_ Makes sense some of the side effects that emerged with $LLY s (e.g. DRG issues) havent been seen to date with $PASG s early data so perhaps will prove to be an additional point of differentiation. Though AAV always risky" [X Link](https://x.com/medstudentinvst/status/1984670515122868315) 2025-11-01T17:14Z [----] followers, [---] engagements "This looks suspiciously like an unattributed rip-off of the hypothesis I articulated across a number of earlier posts including in a comment on this guys own post stating his initial view was that transient LNP / cas9 immunogenicity was the cause of the DILI. If thats true ironic considering our only prior interaction was him taking issue w/ the clear reality that $CRSP will try to argue SyNTase was novel on the basis of non-PACE development and the use of synthetic nucleotides despite it being an obvious rip-off of prime editing. Wherever he got the neo-epitope idea from his proposal for an" [X Link](https://x.com/medstudentinvst/status/1985157992401031504) 2025-11-03T01:32Z [---] followers, [----] engagements "I do grasp what you are saying. I understand this is not for all patients. Indels are stochastic and only a couple out of hundreds had a clinically observable issue. It doesnt matter. If DILI is a risk doctors will go with nucresiran. It would be unethical to do otherwise assuming equal efficacy If you want to bank on HAE more power to you" [X Link](https://x.com/medstudentinvst/status/1985178584474874267) 2025-11-03T02:53Z [---] followers, [---] engagements "Agreed the cause is still unknown and any outcome is possible. However its actually far more complicated than just a simple sequencing of the TTR gene. You first have to take a liver biopsy to even attempt to sequence the TTR gene. Probably dont want to do that to a patient who just met Hys Law. Even if you could take a biopsy you have to keep the biopsy small and hope that sufficient liver cells captured in the biopsy have both been edited and been able to survive any immune attack apoptotic or toxic mechanism. If that doesnt happen the cells wont exist in the biopsy to be sequenced. Amongst" [X Link](https://x.com/medstudentinvst/status/1985264952240648440) 2025-11-03T08:37Z [---] followers, [---] engagements "I have a feeling this $QURE issue may not last overly long You never know. However if there was ever a disease that warranted a right to try Huntingtons Disease is it and the President has consistently backed the right to try. I also dont think Trump will remain uninvolved if the HD community starts getting vocal (as they likely will)" [X Link](https://x.com/anyuser/status/1985336756779032774) 2025-11-03T13:22Z [----] followers, [----] engagements "Okay this is just a musing and Im not saying this is the case. So dont go buying a bunch of $QURE on the basis of this However does the language of the $QURE press release not suggest its possible $QURE may be playing hardball with the FDA / Vinay here Perhaps the meeting with the FDA was more contentious than theyd like Vinay threw his weight around and theyre trying to leverage public pressure (as was seen with Elevidys DMD patients and Sarepta) to make sure they get the outcome they want Basically a jump the chain of command move to get to Marty / RFK or the President who all clearly" [X Link](https://x.com/anyuser/status/1985375253866942661) 2025-11-03T15:55Z [----] followers, [----] engagements "Needless to say the $QURE news has read through to $PASG. Just so anyone who read my post about investing in it is also aware of this new risk / downside. I plan to continue to hold as I believe its FTD-GRN therapy's profile and current market cap continue to present impressive risk-reward regardless of the ultimate confirmatory trial structure / pathway. Recall that $LLY brough proclaim for $880M plus a CVR and $QURE is still worth $1.89Bn. I also think we're a long way from the conclusion of this $QURE story. However I would be remiss not to acknowledge this and the risk definitely just" [X Link](https://x.com/medstudentinvst/status/1985382743845331319) 2025-11-03T16:25Z [----] followers, [----] engagements "I also bought more today so do with that what you will" [X Link](https://x.com/medstudentinvst/status/1985386477174603991) 2025-11-03T16:39Z [----] followers, [---] engagements "@OppInv More $PASG :)" [X Link](https://x.com/medstudentinvst/status/1985395336681001032) 2025-11-03T17:15Z [----] followers, [---] engagements "The $QURE news is actually less of a read through to $PASG than I initially feared based on this. The FDA appears to be taking issue with a failure by $QURE to define the statistical analysis plan (SAP) before study initiation. While I do think HD patients deserve the right to try with AMT-130 as soon as feasible given the severity of the disease and the lack of therapeutic alternatives and $QURE seems to have a legitimate claim that the FDA is flip-flopping in its stance asking for SAP to be defined ahead of trial initiation is actually quite a reasonable stance in most situations otherwise" [X Link](https://x.com/medstudentinvst/status/1985444565046693954) 2025-11-03T20:30Z [----] followers, [----] engagements "November 3rd [----] Daily Update. Added a little bit of new capital into the portfolio to be able buy some more $PASG. Return today before capital addition was 52.5%. Return after new capital is 51.3% as of course the "starting capital" denominator went up and new capital has an effective return of 0%. The latter return is listed. I have posted my existing thoughts on the $QURE pullback today elsewhere. I plan to hold. The portfolio level thing I have to figure out post the $QURE news is whether this portfolio remains appropriately weighted from a risk perspective. I had marked $QURE as a" [X Link](https://x.com/medstudentinvst/status/1985479545642795219) 2025-11-03T22:49Z [----] followers, [----] engagements "Today is so exemplary of just how wild biotech can get. $QURE multi-billion dollar company and media darling down over 50% after basically everyone (few notable exceptions) thought it was an smooth road to BLA submission and approval $SRPT was worth over $15Bn in [----] now down in the [--] billion range Not for the faint of heart boys and girls" [X Link](https://x.com/anyuser/status/1985484124165898658) 2025-11-03T23:07Z [----] followers, [----] engagements "Literally every investor in biotech at some point (probably will be me after $RARE tonight) The pre / post market FUCK is just so real ðŸ˜ðŸ˜”😂" [X Link](https://x.com/anyuser/status/1985726820948361535) 2025-11-04T15:12Z [----] followers, 196.3K engagements "comedic gold @MartinShkreli" [X Link](https://x.com/anyuser/status/1985729270107222172) 2025-11-04T15:22Z [----] followers, [----] engagements "All the idiots buying bitcoin fail to realize that $VKTX is the real store of value. Hashtag uncorrelated asset" [X Link](https://x.com/medstudentinvst/status/1985737087614926994) 2025-11-04T15:53Z [---] followers, [---] engagements "@grok @GeneInvesting Thank you Grok" [X Link](https://x.com/medstudentinvst/status/1985754961695670494) 2025-11-04T17:04Z [---] followers, [--] engagements "@GeneInvesting @grok Oh Tony youre adorable Here it comes. the all-mighty PATIENT SUBTYPPPINNGGGG I get more excited for the full thread by the minute" [X Link](https://x.com/medstudentinvst/status/1985755587477471582) 2025-11-04T17:06Z [---] followers, [--] engagements "Tony (fuming he doesnt know MHC and HLA are the same thing): I dont read your posts man Also Tony: Ive commented on your posts like [--] times in the last couple of days Did you rip-off the I dont read your posts line off from someone too Or did you ask Grok for ideas on how to deflect when your takes have been disproven Now talk to JLaw" [X Link](https://x.com/medstudentinvst/status/1985784483493126318) 2025-11-04T19:01Z [---] followers, [--] engagements "November 4th [----] Daily Update The beatings shall continue until morale improves. Biotech getting absolutely whacked today. Every position I hold was down except $PEPG (). which came in at a really good 0% return for the day. It sure would have been nice to start entering those $RCKT / $PASG positions a few days later.but c'est la vie. $RARE got whacked too after hours on revenue and EPS misses. So I except tomorrow could be bloody. However whilst it's not ideal to lose Crysvita revenue as part of the North American Royalties sale to OMERS they announced this evening it does allow $RARE to" [X Link](https://x.com/anyuser/status/1985859088727548333) 2025-11-04T23:57Z [----] followers, [---] engagements "Good info. Thanks to the OP. My takeaways from this for a registrational study for $PASG s PBFT02 would be: [--]. Include a quantitative volumetric MRI analysis as an endpoint (MOST CRITICAL) [--]. Easier to run an RCT ( if FDA requires) with PBFT02 as the surgery is much less invasive than $QURE s. $PASG s is more like a lumbar puncture [--]. Extra critical to align with FDA (not that this appeared to help with $QURE) on endpoints in non-RCT setting as CDR-FTLD is more susceptible to test administrator subjectivity / bias (i.e. less objective measures) [--]. CDR-FTLD is less susceptible to practice" [X Link](https://x.com/medstudentinvst/status/1985880652940554317) 2025-11-05T01:23Z [----] followers, [----] engagements "I would really encourage retail investors to exercise caution listening to this guy's statements on $NTLA. He has every right to express himself. However you should know he is carrying a very large bag he has pretty consistently demonstrated he is not a good-faith actor in discussions on $NTLA - often failing to meet the baseline standard of accurately representing the substance of opposing views and is willing to use scientific concepts and terminology he appears not to fully understand to justify what is clearly a preordained conclusion that " it was the right move to put 100% of my money" [X Link](https://x.com/anyuser/status/1986168884198596752) 2025-11-05T20:28Z [----] followers, 14.7K engagements "One last thought that is actually to $NTLA 's benefit though definitely wouldn't hang your hat on this. Just one of the first approaches I'd take if I was in charge of trying to solve this problem at the company. The HLA screening idea Tony put forward almost certainly won't work to eliminate DILI risk if the peptide immunogenicity hypothesis is true. As said there are way too many HLA variants and elements that interact with HLA to mediate immunogenicity in each patient and nex-z needs to be able to conclusively rule out DILI risk in patients if its going to compete against the likely" [X Link](https://x.com/anyuser/status/1986168887625252907) 2025-11-05T20:28Z [----] followers, [----] engagements "November 5th [----] Daily Update Didn't get quite as cranked today as I feared with $RARE rebounding a good bit from its after-hour lows yesterday. Think the market realized that non-dilutive financing to support setrusumab (and others in the pipeline) may actually be a good thing for shareholders. Setrusumab critical though especially as baseline revenue goes away with the OMERS deal. I think the $NKTR and $ABVX later buy positions will rebound with time. Not too worried about those $QURE is just ugly at the moment. Got worked again today after the biohaven news. I thought $PASG might hold up" [X Link](https://x.com/medstudentinvst/status/1986258988195807488) 2025-11-06T02:27Z [---] followers, [---] engagements "@david_kochman What makes you say that" [X Link](https://x.com/medstudentinvst/status/1986528275426942979) 2025-11-06T20:17Z [---] followers, [--] engagements "In just awful awful awful news the DILI patient passed away. My thoughts and prayers are with this patient may he rest in peace and his family. I believe this further raises the probability that stochastic CRISPR-cas9 indels / frameshifts can lead to either toxic mRNA or an mRNA that is able to break through nonsense mediated decay and create either a toxic or immunogenic protein. I link to a post I made where I lay out this mechanism in the second post down in this thread. This has a good chance of signaling the end of first-gen CRISPR cas9s viability. If the issue is NMD breakthrough on a" [X Link](https://x.com/anyuser/status/1986555009895432567) 2025-11-06T22:03Z [----] followers, [----] engagements "Dude in good faith if youre in $NTLA I would get out. I dont think you understand the system yet. The primary goal of inducing NHEJ through cas9s DSB in a gene is to create indels that will lead to the genes mRNA being degraded through NMD. The DILI is more likely an on-target effect than an off-target and if it is given a patient died not at all safe" [X Link](https://x.com/medstudentinvst/status/1986620612823896498) 2025-11-07T02:23Z [---] followers, [---] engagements "November 6th [----] Daily Update Getting cooked for real this week. Luckily think $NKTR will see a nice rise tomorrow based on after-hours response to earnings and perhaps see more after the weekend. Single arm rare / severe disease gene therapies ( $PASG $RCKT) getting beat down post $QURE. $QURE news really rocked the thesis that the FDA would be maximally constructive in those spaces. $PEPG dipped about the lowest it has since data. $PRME doing the old plummet too probably mediated by sector effects of $NTLA. Though as said think the most likely causative feature of $NTLA's editor cas9 DSB's" [X Link](https://x.com/anyuser/status/1986626349717434853) 2025-11-07T02:46Z [----] followers, [----] engagements "I really couldn't tell you. No new news. It's seemed to hold the line at $4.50 since 15mg / kg data release in the fall. It jumped up again after the $RNA buyout but figured it would come down after that as I think a buyout is just too abstract at this stage for the market. Perhaps its just low volume. They have two investor conferences next week and they have a real shot at best in class based on early data so maybe that will give it a boost. Then MAD 5mg / kg functional data is likely the next significant catalyst" [X Link](https://x.com/medstudentinvst/status/1986629326255595669) 2025-11-07T02:58Z [---] followers, [---] engagements "Its a very good question. The cas9 nuclease $NTLA uses (as does $CRSP and $EDIT) creates a double strand break in the DNA which results in the endogenous non homologous end joining repair mechanism getting activated and that repair mechanism usually ends up inserting or deleting bases at the site of the double strand break. This is a design feature of cas9 rather than a bug. The number of bases typically inserted or deleted are stochastic. Its most often 1-2 bases but can range to 50+ bases. That will typically cause a frameshift and lead to an mRNA transcript that codes for a very different" [X Link](https://x.com/medstudentinvst/status/1986745377828000207) 2025-11-07T10:39Z [---] followers, [---] engagements "I've said it before and I'll say it again $XBI" [X Link](https://x.com/anyuser/status/1986851804991115629) 2025-11-07T17:42Z [----] followers, [---] engagements "This papers finding (link in comments) that cas9 induced double strand breaks drives a p53 mediated DNA damage response which yields durable markers of cellular senescence and inflammation represents another category of concern for first generation CRISPR and by extension $NTLA and $CRSP. Existing evidence makes it reasonable to think DSB effects both those described in the literature and those undescribed may have consequences that take years or decades to emerge. The extent to which these long-term effects have clinically relevant consequences for older patients with diseases like wild-type" [X Link](https://x.com/anyuser/status/1987836505247604848) 2025-11-10T10:55Z [----] followers, [----] engagements "This is legitimately impressive and unprecedented efficacy data in ATTR-CM from $NTLA. If it were not for the DILI case recently reported $NTLA's stock would have traded up enormously on this news and I think all the reasonable bears would acknowledge this. If acoramidis and vutrisiran were the only game in town $NTLA might have a shot at a viable path forward on the heels of this data pending the outcome of the DILI investigation. Heart failure is a big deal and obviously kills people. So there is an ethical and rational basis for prescribing a drug that has very rare cases of DILI if it" [X Link](https://x.com/anyuser/status/1988002657043390800) 2025-11-10T21:55Z [----] followers, [----] engagements "Definitely possible. I really don't have any insight into the current trading dynamics. From my perspective $PEPG still is in possession of best-in-class data for a DM1 asset at this stage the $RNA buyout was a good comp for $PEPG's upside if they can continue to demonstrate best-in-class data and I have not seen anything to warrant a change in my view at this time. However we'll see" [X Link](https://x.com/medstudentinvst/status/1988003173597147303) 2025-11-10T21:57Z [---] followers, [---] engagements "@AlexHumphries25 How are you going to definitively mitigate stochastic cas9 indels that create toxic mRNA or mRNA that breaks through nonsense mediated decay and yields an immunogenic peptide" [X Link](https://x.com/medstudentinvst/status/1988019277711241515) 2025-11-10T23:01Z [---] followers, [---] engagements "Certainly fair to point to the fact this is Phase I open-label data. However there are few unique characteristics of ATTR which I think allow one to reach stronger conclusions on efficacy on the basis of Phase [--] data than one could in other diseases. First the dissolution of the TTR tetramer into monomer and then aggregation into amyloid is about as definitively established as the proximal monocausal mechanism of TTR cardiac amyloidosis as it can get in medicine and without TTR it really shouldn't be possible to get the amyloid build-up. Two we have really definitive data from tafamidis vs." [X Link](https://x.com/medstudentinvst/status/1988068140031311932) 2025-11-11T02:15Z [---] followers, [---] engagements "Thank you for the response The preclinical studies unfortunately wouldnt be able to rule that out especially because animals have a different TTR sequence than humans so an indel induced frameshift in them doesnt generate the mRNA sequence that gets generated from an indel induced frameshift in humans and wouldnt generate the same protein. Humans also have quite a bit of TTR variation over [---] variants" [X Link](https://x.com/medstudentinvst/status/1988180678383435935) 2025-11-11T09:43Z [---] followers, [--] engagements "@BK7959925597715 Dude sorry I missed this. I know it is definitely a strange market quirk that a fire sale would almost certainly fetch well north of where it currently trades. One argument for early stage companies staying private for longerthough we wouldnt get access" [X Link](https://x.com/medstudentinvst/status/1988210743427428801) 2025-11-11T11:42Z [---] followers, [--] engagements "@BiotechObserver Its because nex-zs TTR knockdown happens much quicker and is deeper than vutrisiran or patrisiran. $ALNYs next-gen RNAi in development (nucresiran) has shown equivalent depth and speed of TTR knockdown in humans so likely will show same efficacy as nex-z" [X Link](https://x.com/medstudentinvst/status/1988279725123661876) 2025-11-11T16:16Z [----] followers, [---] engagements "@Alex___VA Yes for him I certainly agree he is disputing the efficacy. Based on the comments Ive seen I would not include him in the category of $NTLA bears who are well-informed or logical about the situation" [X Link](https://x.com/medstudentinvst/status/1988340794953232664) 2025-11-11T20:19Z [---] followers, [---] engagements "Me looking at green when Ive developed Stockholm syndrome for red" [X Link](https://x.com/medstudentinvst/status/1988350112683450405) 2025-11-11T20:56Z [---] followers, [---] engagements "November 11th [----] Daily Update Sure do love FDA rumors. 9.6% jump on the day. We'll see if it holds $XBI" [X Link](https://x.com/anyuser/status/1988355302748512629) 2025-11-11T21:17Z [----] followers, [---] engagements "Little surprised I'm not seeing as much chat on $PCVX as I would have expected though perhaps it's just lost in the algorithm. It has run some from its lows but been an attractive opportunity imo" [X Link](https://x.com/medstudentinvst/status/1988356878925971636) 2025-11-11T21:23Z [----] followers, [---] engagements "November 12th [----] Daily Update BIG jump for $PASG off its recent lows. It is of course a high risk investment and may remain volatile through the 1H [----] updates. However I continue to believe the risk-reward profile here is extremely strong. To that point $LLY paid $1.04Bn (inclusive of a CVR) for Prevail and its FTD-GRN therapy back in [----] both $PASG and $LLY are in Phase [--] / [--] (though should note Prevail also had a PD asset in clinical development) and my view is that $PASG has shown more compelling data to date on both efficacy and safety. $PASG is still trading under $30M at the" [X Link](https://x.com/anyuser/status/1988722338196787622) 2025-11-12T21:35Z [----] followers, [----] engagements "November 14th [----] Daily Update Little delayed in the Friday daily update post. On Friday I partially exited the $BBIO position and reallocated the proceeds towards $PRME. I will finish up a post tonight or tomorrow detailing my rationale but wanted to get this out before markets reopen for the public record" [X Link](https://x.com/anyuser/status/1990179020319670480) 2025-11-16T22:03Z [----] followers, [----] engagements "I have increased my $PRME position for five reasons. First and foremost I believe it is highly likely that PM359 ( $PRME's CGD therapy) will receive FDA approval under the plausible mechanism pathway in the coming months as it definitively meets each of the five tenets of the FDA's plausible mechanism pathway. Indeed if one were to write an article explaining the rationale for approving CGD on a standalone basis I think it would look almost identical to the article Dr. Makary and Dr. Prasad published. Furthermore there are a number of pieces of circumstantial evidence that lend credence to" [X Link](https://x.com/anyuser/status/1990406561219383679) 2025-11-17T13:07Z [----] followers, 11.4K engagements "November 17th [----] Daily Update. $PRME had a nice rally into the close following the increase in position size on Friday. I explained why I increased the position in a separate post. We will see if it holds. $PEPG also showed real strength today. $NKTR did as well. $ABVX continues to inch up. These four drove a gain today in the portfolio. Also opened a very small position in $IVVD at pre-market pricing. Lastly sold some more of the $BBIO position. It's a challenge for me to prioritize between positions I think are attractive amidst a finite pool of capital. I still think $BBIO can run." [X Link](https://x.com/medstudentinvst/status/1990577297653473566) 2025-11-18T00:26Z [---] followers, [----] engagements "This is a very good post by Adam. I'd like to add a couple of thoughts on AMT-130 and speak to the applicability of some of these concepts to $PASG and alpha-synuclein targeted therapies in Parkinson's Disease. First there may be an issue with tominersin in addition to the ones Adam spoke to. The huntingtin protein is absolutely essential to neuronal (and glial for that matter) function and health. This is well described in both human beings and animal models. In fact human beings born with compound heterozygous mutations in the HTT gene (one mutated copy from each parent) experience a rare" [X Link](https://x.com/medstudentinvst/status/1990608958600380685) 2025-11-18T02:32Z [----] followers, 14.1K engagements "$PASG has published data on the biodistribution of the AAV1 Vector / PGRN transgene in their animal models which should correlate to PGRN expression unless theres a question around whether the transgenes are actually driving expression. My guess is youre referring to the [----] study out of Penn. That study actually showed AAV1 transduction was widespread throughout the brain though only transduced about 1% of neurons astrocytes etc. deep to the ependymal cells in NHPs. So youre right that the meninges / ependyma are transduced in higher quantities and concentrations of PRGN in the CSF are" [X Link](https://x.com/medstudentinvst/status/1990728243071054094) 2025-11-18T10:26Z [----] followers, [---] engagements "Maybe Im misunderstanding you but what do you think is nonsense speculation here I think the vast majority of commentary here is evidenced based and any speculation is both caveated as such and reasonable. I also do think this is an important conversation. I dont see how theres any way to say that AMT-130 doesnt work and to my eye there is more evidence than any other HD therapy has shown previously that it may work. So whether current HD patients have the right to try it on the basis of existing data I think is a conversation worth having irrespective of any investment considerations. i" [X Link](https://x.com/medstudentinvst/status/1990735469160612193) 2025-11-18T10:54Z [---] followers, [----] engagements "Regarding $LLY theyre in Phase I and havent shown any human data at all yet nor substantive preclinical data. If youre concerned about $PASG not publishing ISF PGRN concentrations (just vgs) you should be particularly concerned about $LLY / Prevail in that respect. As an additional point re human data it is notable that we know of no living human with a high-confidence LoF alpha-synuclein variant which precludes the kind of MR studies that have shown benefit in most other siRNA contexts. Further studies show that in active PD outcomes are worse for patients who have lower levels of monomeric" [X Link](https://x.com/medstudentinvst/status/1990767168238051342) 2025-11-18T13:00Z [----] followers, [---] engagements "You seem to believe a PBFT02 failure is fait accompli though the basis for that appears to center on the fact that $PASG has not reported PGRN ISF concentrations that appears you presume they are hiding. It would be rather strange they would take the low-dose cohort forward if there is some unreleased internal data suggesting that is an issue. They will start to report more fulsome data in 1H [----] so we will see how that unfolds. The data from the new cohort of patients they dosed very early in disease course will be especially interesting. Whilst I fully acknowledge the high risk nature of" [X Link](https://x.com/medstudentinvst/status/1990797447321337935) 2025-11-18T15:01Z [----] followers, [---] engagements "Correct re spreading occurring. However this is where some nuance comes into play. This is actually a very different phenomenon in $QUREs case and may be a good thing as early HD pathophysiology might be broader in site of origin that just the striatum). The key thing to see with $QUREs is that profile of spread from the site of injection in the striatum is going to be a precise dose delivered to the striatum at the level you think will knock down mHTT sufficiently and then the levels of miRNA expressed will get lower as the AAV spreads out from the site of injection. So youre titrating the" [X Link](https://x.com/medstudentinvst/status/1990849758395560306) 2025-11-18T18:29Z [----] followers, [---] engagements "Whatever your views on $QURE this is actually a very important point. Whilst it is foolish to think experts don't get things wrong all the time or that a lay person who is smart and reads up on a topic can't come up with far better ideas than the so-called experts.it is similarly foolish to think the average doctor or average neurologist possesses anything approaching the breadth of HD specific domain knowledge or intellectual horsepower of Dr. Tabrizi. She is rightly considered a genuine leader in the HD field Doesn't mean she is guaranteed to be right here and differing viewpoints should be" [X Link](https://x.com/medstudentinvst/status/1990935336831025337) 2025-11-19T00:09Z [----] followers, 19.1K engagements "November 18th [----] Daily Update $PEPG continues to rip on though backed off some at the end of the day. $PRME comes back down from the end of the day jump yesterday" [X Link](https://x.com/medstudentinvst/status/1990936319099281870) 2025-11-19T00:13Z [---] followers, [----] engagements "I think an HD patient has the right to make that call for themselves under informed consent. I am not so sure you would have the same views on this if you were staring down the certainty of a horrible loss of your memory reasoning capabilities motor function and ability to take care of yourself leading to an early death. If I were an HD patient and at an age where I was likely to convert from asymptomatic to symptomatic I would personally choose taking this shot. I will note one of those three patients had an CSF leak due to an LP required as part of the trial to do assays on the CSF." [X Link](https://x.com/medstudentinvst/status/1991103637511094337) 2025-11-19T11:17Z [---] followers, [---] engagements "Added remaining cash from exits to $JANX. Janx now about 5% of portfolio" [X Link](https://x.com/medstudentinvst/status/1995518352253685907) 2025-12-01T15:40Z [----] followers, [----] engagements "Further exited the $RARE position to add to $JANX. Full breakdown posted tonight. However want to publicly disclose for accountability given post-market update" [X Link](https://x.com/medstudentinvst/status/1995527227501314376) 2025-12-01T16:15Z [----] followers, [----] engagements Limited data mode. Full metrics available with subscription: lunarcrush.com/pricing
@medstudentinvst WaveStrum Life Sciences
WaveStrum Life Sciences posts on X about $prme, $ntla, $pepg, in the the most. They currently have [-----] followers and [---] posts still getting attention that total [---] engagements in the last [--] hours.
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- [--] Week [--] +41%
- [--] Month [--] -7.50%
- [--] Months [---] +2,777%
- [--] Year [---] +37,500%
Followers: [-----] #
- [--] Week [-----] +1.30%
- [--] Month [-----] +3.60%
- [--] Months [-----] +2,793%
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Social Influence
Social category influence stocks finance cryptocurrencies technology brands countries currencies travel destinations
Social topic influence $prme #14, $ntla, $pepg #7, in the, update, daily, $nktr #25, $qure, $crsp, $pasg #7
Top accounts mentioned or mentioned by @geneinvesting @seedy19tron @zhaoweiasu @endpts @martinshkreli @drjesstaylor @plainyogurt21 @aditharun @grok @amaymd @capitalshipyard @winklevosscap @biopharmiq @melvinriskmgmt @cdcgov @capitalusher @stmkrs @cernovich @monacobiotech @hall8jack
Top assets mentioned Prime Medicine, Inc. (PRME) Intellia Therapeutics, Inc (NTLA) PepGen Inc. (PEPG) uniQure N.V. (QURE) CRISPR Therapeutics AG (CRSP) Passage (PASG) Abivax SA (ABVX) SuperRare (RARE) Vaxcyte, Inc. (PCVX) Avidity Biosciences, Inc. (RNA) Alnylam Pharmaceuticals, Inc. (ALNY) BEAM (BEAM) Wave Life Sciences Ltd. Ordinary Shares (WVE) Arrowhead Research Corporation (ARWR) BridgeBio Pharma, Inc. Common Stock (BBIO) Eli Lilly and Company (LLY) Janux Therapeutics, Inc. (JANX) Verve Therapeutics Inc. (VERV) Viking Therapeutics, Inc (VKTX) The Pink Panther (PRIME) Viking Holdings Ltd (VIK) Metagenomi Technologies, LLC (MGX) NVIDIA Corp. (NVDA)
Top Social Posts
Top posts by engagements in the last [--] hours
"$BBC's decline of -43.43% since August [----] relative to $SPY's gain of +90.91% shows just how poorly the biotech sector has performed in terms of valuations and underlying capital outflows. This has occurred amidst massive upwards pressure on equity values from inflation in general (+24ish% since 8/2020) and enormous M2 expansion in specific (up more than 40% from the start of 2020) tempered marginally by a rising rate environment. Significant renewed interest in the biotech sector and significant renewed capital inflows from non-life science asset managers prospective life science LPs and"
X Link 2025-08-13T17:49Z [--] followers, [--] engagements
"$PEPG 13F Updates Q2 [----] Top [--] Investors (45.5% of shares outstanding): [--]. RA Capital: [--------] shares (HOLD Q1 to Q2 '25) [--]. Point72: [-------] shares (3.1% INCREASE in Q2 '25 from [-------] in Q1 '25) [--]. Viking Global: [-------] shares (HOLD Q1 to Q2 '25)"
X Link 2025-08-14T21:07Z [--] followers, [---] engagements
"$PEPG 13F Updates Q2 [----] Top [--] Institutional Investors (45.5% of shares outstanding): [--]. RA Capital: [--------] shares (HOLD Q1 to Q2 '25) [--]. Point72: [-------] shares (3.1% INCREASE in Q2 '25 from [-------] in Q1 '25) [--]. Viking Global: [-------] shares (HOLD Q1 to Q2 '25)"
X Link 2025-08-14T21:13Z [---] followers, [---] engagements
"August 15th [----]. Small adds to $RARE and $NTLA today and doubled the size of $NKTR position. $NTLA position getting very close to maximum sizing I am comfortable with. $RARE starting to get close as well. A substantial percentage of available capital has now been deployed. This fall will have a lot of key data readouts for the portfolio. Key task this weekend is to make a decision on whether to hold or sell $ALNY"
X Link 2025-08-16T03:59Z [----] followers, [----] engagements
"For whatever it is worth the enrollment rates in nex-z's Phase III trial is the best empirical evidence we have regarding the degree of patient and physician demand for nex-z especially as enrollment is occurring at the same time as acoramidis's and vutrisiran's commercial ramp and nucresiran entering Phase III. The fact $NTLA expects to enroll [---] patients by year end (first dose in 2024) is a reasonable indication demand exists. Just [---] patients a year in commercial stage would almost certainly get $NTLA to at least a $1.77Bn EV from its current $1.18Bn (conservatively: $1.5M price *4.5x"
X Link 2025-08-17T18:21Z [--] followers, [--] engagements
"You are missing the critical statistic. What percent of the aggregated ceiling value of the contracts in each size bucket is spent Or provide a bar graph showing the percentage of contracts within different buckets of percentage of ceiling value spent. If 38% of contracts over $1Bn were spent to ceiling and 61% of $1Bn+ contracts were spent to say 98% of ceiling value your graph would presumably still just show that 38% even though 99% of all $1Bn contracts are effectively spent to ceiling. That would be highly misleading. I dont know what percentage of aggregated ceiling contract value gets"
X Link 2025-08-18T11:32Z [--] followers, [--] engagements
"$CRSPR in vivo is basically just first-gen CRISPR's version of $VERV with a dash of a permanent version of $ALNY / $ARWR cardiovascular and AHP. Will say Lp(a) is a massive opportunity that I don't think is fully appreciated. Population wide testing and treatment should become standard of care within the next decade and will produce at least one blockbuster maybe more. [--]. 20%-30% of population have elevated levels [--]. Biggest inheritable risk factor for CVD which is the leading cause of death in the US and globally [--]. Risk is cumulative and driven by number of years of exposure so early"
X Link 2025-08-18T14:35Z [--] followers, [---] engagements
"Prime Medicine - $PRIME. Potential to cure (not just treat) 90% of genetic diseases and transform medicine in unprecedented way. Somewhat derisked as $VERV bought out for $1Bn and has MC of $471M. Nobel Prize level science. Technology out of Broad / Harvard / MIT. Alphabet largest institutional investor"
X Link 2025-08-18T22:56Z [--] followers, [--] engagements
"The answer is compound growth. $PRME is effectively a venture play in a company that happens to be publicly traded. Lets say $PRIME becomes a $50Bn company if successful. I think anyone who is an investor at this stage has to be thinking $PRMEs upside case is at minimum near $ALNYs valuation. That would be about 100x from current prices. Investing now at $3.50 vs $7.00 a year down the line may not seem like much of a difference. However that would halve a 100x return to 50x. Similarly investing at $1.75 a few weeks ago would have made the return 200x at the same $50Bn EV. Thats a ton of"
X Link 2025-08-19T03:49Z [----] followers, [----] engagements
"Yes its certainly true if $NTLA goes up 4x in the timeframe it takes $PRME to go up 3x one can get an additional 1x of return to invest back to $PRME. There are two challenges with that. First I dont think that scenario is by any means guaranteed because compound growth cuts in favor of $PRME in a second way. $NTLA going up 4x ($1.15Bn to $4.6Bn) requires an additional $3.6Bn in value creation in the markets eyes. $PRME going up 4x ($500M to $2Bn) in the same timeframe only requires an additional $1.5Bn. The market could read that into $PRME merely by starting to believe $NTLA and gene"
X Link 2025-08-19T05:35Z [--] followers, [---] engagements
"It certainly could play out that way. If it does I have a good size stake in $NTLA and would be happy with the returns there. However if it doesnt perhaps bc $PRME starts getting valued off the $VERV buyout price which would get it to 4x from here alone probably whilst nucresiran shows strong results and the market grows increasingly wary of $NTLA I still have a stake in $PRME didnt miss out on the extra 100x / 200x (or more) if $PRME proves successful by getting in at a marginally higher price nor is the $PRME investment predicated on the $NTLA investment if a tail risk event happens and"
X Link 2025-08-19T06:10Z [--] followers, [---] engagements
"I do not think it is true being [--] years ahead makes it hard for $BEAM's valuation to struggle if $PRME has a superior therapy with no bystander edits. There's a lot of historical precedent in biotech that a superior therapy depresses a competitor's valuation in the market's eyes even if it's a couple years behind. This is further true if $WVE proves to also have a superior drug to $BEAM as they are closer than $PRME and would allow a number of patients to bridge to $PRME if they wanted the gene editing solve. Yes nucresiran could only show results in [--] years and $NTLA could have [--] approvals."
X Link 2025-08-19T12:49Z [--] followers, [---] engagements
"August 19th [----]. Exited 50% of $ALNY position for a +94.1% gain. Exited 2/3 of $VKTX position for a +1.9% gain. Remaining 1/3 is down -0.3% after the events of the day. $ALNY - This very much highlighted to me the challenge of investing in the really large biotechs as an individual investor with other professional commitments. I developed significant conviction $ALNY was undervalued based off of the ATTR pipeline and other approved therapies. However $ALNY 's pipeline page has [--] indications. Diligencing the competitive landscape the therapeutic's mechanism of action the market size etc. for"
X Link 2025-08-20T06:22Z [--] followers, [---] engagements
"Jane Street is a quant fund. They are not taking this position because they have a view on the long-term value of the $CRWV. They don't invest based on that. Their algorithms take positions (usually very short-term positions) in companies when signal(s) appear that have a certain probability of being followed by a certain short-term price movement as identified by statistical analysis of massive historical datasets. If you're making this claim from a 13F filing or the like there is a good chance they have already traded out of the position"
X Link 2025-08-20T23:04Z [--] followers, [---] engagements
"August [--] [----] Continued to add to $ABVX and $NKTR. Cost basis moves slightly up for both. As per my post earlier would be a real win if $PRME is able to secure CGD approval. Going to be an important fall / winter for the $NTLA $PCVX and $RARE positions. Very interested to see the data from $PEPG this October. $BEAM and $MGX are my lowest conviction positions at the moment in terms of the long-term competitive advantage / commercial prospects of their pipelines. I am going to continue to hold for now given small position size the demonstrated efficacy of base editing and as a diversification"
X Link 2025-08-21T21:07Z [----] followers, [----] engagements
"42% of companies in the Russel [----] are not profitable. One 25bps rate cut is not going to change that reality nor will it automatically change the reality of mortgage rates debt financing costs etc. So on that view it is strange to see the Russel [----] up 4% today. Now with that said the prices of investable assets move off each market participant's individual buy and sell decisions in response to their individualized moment by sentiment about an asset's forward looking prospects - sentiment defined as the combined effect of reasoned conclusions intuition and emotions So if one rate cut"
X Link 2025-08-22T17:13Z [--] followers, [---] engagements
"Speaking of sentiment shiftthe market participants in which a sentiment shift matters most for biotech investors is the $LLY s $AMGN s $GSK s etc of the world. If their sentiment shifts towards the belief that biotech valuations will start to run significantly it likely accelerates and compresses any M&A activity. Imagine explaining to your board you missed out on getting a highly promising acquisition target on the cheap in one of the worst biotechs markets of all time because you were too slow Seeing significant M&A activity would of course be an additional accelerant to the broader markets"
X Link 2025-08-22T19:17Z [---] followers, [---] engagements
"August 25th [----] $NTLA - nice to see a little bit of a bump due to Friday's insider buy on a day $XBI and $BBC were down 2.5%. $CRSP - Initiated a small $CRSPR position and will continue to add some more over the coming weeks. Target is about [--] to 4% of the current portfolio. This was a tough investment for me to make in a lot of ways. I am not super excited about the commercial prospects of Casgevy (especially with the [--] / [--] rev share with $VRTX) or the nature of the therapy itself - chemo just sucks for patients though recognize the trade off is worth it for some SCD patients. I think in"
X Link 2025-08-25T22:42Z [----] followers, [----] engagements
"August 26th [----] Realized after my post yesterday a larger $CRSP limit order ended up going through earlier in the day. risks of tracking positions on the move / on your phone. So $CRSP position is at 4.5% of portfolio. That is the upper end of the portfolio size I was planning to build to over the next few weeks. So will hold here for now. As said previously the Lp(a) therapy drives my $CRSP investment. Lp(a) data readout in 1H [----] will be key. $CRSP will need to show comparable efficacy to $LLY's siRNA which has had really nice results thus far as it enters Phase III. If $CRSP shows"
X Link 2025-08-27T01:54Z [---] followers, [---] engagements
".even though a tech contraction would probably drive a rotation of capital back to the biotech sector especially if it occurs alongside commercial validation of innovative platform biotechnologies (e.g. gene editing RNAi etc.) If $NVDA misses earnings today were all gonna die If $NVDA misses earnings today were all gonna die"
X Link 2025-08-27T18:16Z [--] followers, [---] engagements
"August [--] [----] $BBIO - my curiosity is always piqued by the underlying drivers of the between news / event price action with BBIO given the KKR Viking etc. presence. However nice to see it find the $50's and hit a [--] week high. If they are able to compete effectively with $ALNY as data on scripts continues to get released there is likely room to run there $ABVX / $NKTR - the de-risked investments post data release have played out pretty well thus far $NTLA - I am not expecting much until the data releases this fall. However good to see a little resilience in the share price though it remains"
X Link 2025-08-28T21:37Z [---] followers, [---] engagements
"August 29th [----] Tough end to the week for $XBI / $BBC 🤣 $NKTR a lone bright spot. Nice to see $NTLA not get crushed every once in awhile on the down days little more resilient these days on the whole I would say. Though important to remember its the long-term that matters and not the day to day price movements. My hope is that pricing stays around these ranges through the historically tough month of September and into the data readouts that will start coming fast and furious in October. $PEPG likely the earliest readout. Have considered buying some downside protection / trying to play the"
X Link 2025-08-29T20:42Z [---] followers, [---] engagements
"Eh I don't think that's true. $74.7M cash as of 6/30/25. Q2 cash burn of $23.1M so $7.7M per month. $18.2M of Q2 burn is R&D and personnel which will be initial cost savings targets in a wind-down. Make or break data comes early Oct' [--]. If $PEPG continues to burn at $7.7M through 11/30/25 that's $38.5M of burn and $36.2M of cash remaining which is just under their current market cap of $38.7M. Critically half of R&D and an unquantified percentage of personnel spend in Q2 was for DMD therapy discontinued on 5/28. So that's already being wound up $7.7M monthly burn is therefore likely already"
X Link 2025-09-01T23:44Z [--] followers, [---] engagements
"Objectively true. In the words of the great Parcells you are what your record says you are. .and these CDC bureaucrats have a record that is abysmal. The arrogance and self-importance of these @CDCgov bureaucrats is beyond belief. The reality is that the US would have been better off with no CDC at all during the pandemic. Their performance was a Chernobyl-level catastrophe. Their incompetence delayed the start of widespread https://t.co/Juzfbs7nmL The arrogance and self-importance of these @CDCgov bureaucrats is beyond belief. The reality is that the US would have been better off with no CDC"
X Link 2025-09-02T00:58Z [--] followers, [--] engagements
"Many are starting to finally wake up to what $LLY and $GOOG realized wrt $VERV and $CRISPR realized wrt to its own pipeline which is that gene editing in CVD will have unprecedented impact on patient health and enormous commercial return so long as no safety events emerge. Latent safety events is a risk impossible to rule out at this point but the risk-reward is astounding. Atherosclerotic CVD is the biggest killer of human beings in the world atherosclerosis builds up from very young ages yet is largely preventable with early lowering of ApoB in a manner that mimics well-studied naturally"
X Link 2025-09-02T14:39Z [---] followers, [---] engagements
"So under that view wise business decision for $ARWR to license that out"
X Link 2025-09-02T14:50Z [---] followers, [--] engagements
"Best case scenario for Novartis is a donanemab like minimal benefit"
X Link 2025-09-02T14:54Z [---] followers, [--] engagements
"$PRME can meet all five elements of the FDA's RDEP criteria with its CGD program. "Approval under the process may be based on one adequate and well-controlled study plus robust confirmatory evidence (☑) which may include: Strong mechanistic or biomarker evidence ☑ Evidence from relevant non-clinical models ☑ Clinical pharmacodynamic data ☑ Case reports expanded access data or natural history studies ☑""
X Link 2025-09-04T01:39Z [----] followers, [----] engagements
"https://www.fda.gov/news-events/press-announcements/fda-advances-rare-disease-drug-development-new-evidence-principles https://www.fda.gov/news-events/press-announcements/fda-advances-rare-disease-drug-development-new-evidence-principles"
X Link 2025-09-04T01:39Z [---] followers, [---] engagements
"September 4th [----] Sold $ARWR for 130.1% gain though a very small part of the portfolio. I believe most of the value of $ARWR is contained within its APOC3 (sHTG FCS especially) and ANGPTL3 pipeline (Dyslipidemia / HoFH). Shame they no longer have the Lp(a) therapy though it looks to be behind $LLY's. The FCS and sHTG directed therapies remain very interesting. I need more time to determine whether current valuations are justified by $ARWR's competitive positioning within the sHTG market. I think the ANGPTL3 therapies are somewhat vulnerable to the one-and-done gene editing therapies but"
X Link 2025-09-04T21:35Z [---] followers, [---] engagements
"This will be my thread for my concerns around the INHBE / ALK7 silencers from $ARWR and $WVE. The primary thesis is as follows: [--]. Insulin resistance is counter-intuitively usually characterized by overly active adipocyte triglyceride lipase and hormone sensitive lipase as insulin no longer has the same inhibitory effect on these enzymes found in adipocytes. This overactive ATGL and HSL leads to a huge excess of free fatty acids (FFAs) released in the plasma. These FFAs cause lipotoxicity in the surrounding cells and amongst many other things drive increasing insulin resistance. [--]. Most obese"
X Link 2025-09-04T22:26Z [----] followers, [----] engagements
"Longer post with some more pathway detail made earlier today: I am concerned about $WVE's INHBE silencer's lipolysis enhancing effects in patients that are already diabetic or obese (not born with a LoF variant from birth) as INHBE silencing's mechanisms of action appear to work in opposition to the lipolysis inhibiting effect of a drug class (PPAR- agonists) shown in humans to be highly effective for improvements in blood glucose and insulin sensitivity in diabetic and insulin-insensitive obese patients. The INHBE pathway has a primary effect of inhibiting lipolysis and is governed by the"
X Link 2025-09-04T22:30Z [----] followers, [---] engagements
"Both $ARWR and $WVE are running trials of their INHBE / ALK7 silencers as a monotherapy in obese patients (see attached photos) and so weight loss using INHBE / ALK7 silencers would appear to remain a key objective for the companies. I also think Bruce Givens left $ARWR in [----]. I think the use of NHBE / ALK7 silencers in formerly insulin resistant / obese patients who have restored metabolic health has a reasonable chance of mirroring the metabolically beneficial effects of the LoF INHBE / ALK7 genetic variants by promoting the maintenance and resilience of a healthy metabolic state with are"
X Link 2025-09-05T21:34Z [---] followers, [----] engagements
"September 5th [----] Fully exited the $ALNY position for a gain of 93.4% today though this was a small position in the portfolio. Nice jump to the portfolio in general today. Though the truth is if I am fundamentally wrong on the therapeutic outcomes / commercial viability of a lot of my biggest positions an uplift in the broader biotech market as was seen today won't really matter. I continued to add to $NKTR and $RNA today which brings the cost basis for both slightly up. I will likely continue to add to both so am honestly hoping for a bit of a dip here. I continue to think the risk-reward"
X Link 2025-09-05T21:46Z [---] followers, [---] engagements
"Yeah this is objectively true. Inner city thugs are fake tough. The coward that murdered Iryna Zarutska in cold blood is an obvious example. Went after a defenseless girl on a bus with a knife They would never go after say a jacked combat veteranespecially if the combat veteran is armed to an equivalent degree. Why Because a) these thugs really are soft to their core and b) they know they would get their heads canoed In the rare event they pull this shit without realizing a red-blooded American male is present its always these losers who die. Just like the one Daniel Penny (certified legend)"
X Link 2025-09-09T12:08Z [---] followers, [---] engagements
"This is rending. Penetratingly horrific. The never ending release of violent criminals MUST NOT BE ALLOWED TO CONTINUE. Every single person in America must buy into the following principle: if you choose to engage in an act violence against an innocent in America you get viciously taken down and put away without apology. This coward was released [--] times. 🚨BREAKING: New footage has surfaced of Iryna Zarutska in the moments after career criminal DeCarlos Brown Jr. stabbed her in the neck. Absolutely infuriating and heartbreaking. This is who they let free [--] times. https://t.co/QziLkGouc3"
X Link 2025-09-09T19:45Z [---] followers, [---] engagements
"@capital_usher A good comp if $PRME is successful is $VRTX. They reached a $100Bn+ market cap on the basis of non-curative CF therapies"
X Link 2025-09-10T17:35Z [---] followers, [---] engagements
"They will need to raise (or find other non-dilutive financing like partnerships though would need major partnerships across multiple indications so unlikely to fully fund CF development) in order to bring CF to market. The hope would be that Wilson's and AATD ultimately provide revenue and bolster valuation in the meantime so that the raises are less dilutive"
X Link 2025-09-10T17:40Z [---] followers, [--] engagements
"@DrJessTaylor Thank you for sharing this message. It is so critical that people of all political viewpoints share the fundamental commitment to the right to the freedom of speech and to the sanctity of innocent human life as you clearly do. Your decency is deeply appreciated"
X Link 2025-09-11T01:33Z [----] followers, [----] engagements
"Let me get this straight. You think someone who advocates for pardoning an individual convicted of a violent offense deserves to be murdered for stating that view Im not going to waste time debating Jan. 6th with you. The evilness of your line of thinking is clear even if Jan. 6th happened the way you suggest"
X Link 2025-09-11T18:18Z [---] followers, [----] engagements
"@Stmkrs Check out a post I made earlier today further down this thread on $ARWR s DIO mouse model. Put a few thoughts about that there"
X Link 2025-09-11T18:41Z [---] followers, [---] engagements
"You are so emblematic of the banality of evil. How is it that the most mediocre and ineffectual of people people that would never stride into danger or demonstrate courage amidst great sacrifice are the ones that celebrate horrible violence the loudest. If you have to lie about Charlies points you dont have a point. Also even if he was someone who made the points you claim words are not violence no matter how much you may desperately wish to remain arrogantly removed from dissenting views. Viciously slaughtering someone speaking peacefully whilst his wife and children are watching is real"
X Link 2025-09-12T11:34Z [----] followers, [----] engagements
"It is good to see that you subtly acknowledge his point was not at all that innocents should be murdered or that the fact they are murdered isnt horribly tragic even if you dont have the honor or stones to come right out and say it or answer the question posed to you. It seems you are now saying a public figures phrasing of a point could have been better though you recognize the validity of his underlying point. I think his phrasing was just fine and he actually had the guts to speak to a brutal trade-off unlike you who is too cowardly to actually take a stance on the question posed to you."
X Link 2025-09-12T12:33Z [---] followers, [--] engagements
"The simple truth is that Charlie Kirk represented the views of around 50% of America on most issues and on many issues (like men in women's sport) far more. Yet huge swaths of the left have declared that if you dare to have these views or most any view different than the leftist orthodoxy you either deserve to die or are not worthy of being mourned. You and your views will also be twisted misrepresented and lied about in a desperate effort to justify explicitly or implicitly your murder. Anyone who has spent any time in academia or around leftists knows how real and widespread this sentiment"
X Link 2025-09-13T11:21Z [---] followers, [--] engagements
"I have not wanted to post about amateur biotech investing since Charlie's murder. However to maintain public accountability of performance for whenever I do go back to doing the daily performance update here are the three days I've missed:"
X Link 2025-09-14T14:11Z [---] followers, [--] engagements
"Why Being Supportive of Employers Firing Individuals Celebrating the Murder of Charlie Kirk is Perfectly Consistent with a Belief in Free Speech It is perfectly consistent to be a staunch advocate for the right to free expression of ideas without government-mandated punishment and also believe that employers have a right to fire employees who express beliefs the employer reasonably believes have a high likelihood of materially harming the employees ability to do the job they were hired to do effectively. This is especially true in professions with the responsibility of providing life-saving"
X Link 2025-09-16T12:53Z [---] followers, [--] engagements
"The sheer number of major voices in the conservative movement immediately and correctly calling out the fact that so-called hate speech is protected by the First Amendment whilst speech that actually threatens plans or intentionally incites violence is not demonstrates how sincerely committed most major figures in the American conservative movement are to free speech. This is a good thing and shows the movement is capable of principled self-governance. Hate speech is not prosecutable in America (which is good). Pam Bondi knows this. I am guessing given the statements by Stephen Miller"
X Link 2025-09-16T13:53Z [---] followers, [--] engagements
"You have enemies Good. That means youve stood up for something sometime in your life - Winston Churchill This quote should always come to mind whenever one see the lies and hate directed towards Charlie. For he didnt just stand up for something. He stood up for the most important things - his faith his family his country and his free thought and speech. Always and relentlessly"
X Link 2025-09-17T01:17Z [---] followers, [--] engagements
"Wednesday September 17th Significant reversal in prices post rate cut announcement. The portfolio got as high as 35% return before coming back down. As always $PRME has heavy influence. My suspicion is it will be challenging for $PRME to move far above $5 in the next couple of months unless an announcement is made about accelerated approval pathways in the context of CGD or its other. However I believe its true present value is significantly higher than its current pricing range so a broader market alignment with that view could always occur and therein drive pricing higher. $NKTR continues"
X Link 2025-09-17T22:11Z [---] followers, [---] engagements
"Candace Owens grift is impressively annoying"
X Link 2025-09-18T01:12Z [---] followers, [--] engagements
"This graph is misleading and much of the analysis here is either inaccurate or incomplete. First the y-axis is showing total reported cases of acute Hep B cases in the US not infant Hep-B cases. I understand you likely misread the title of the graph but it should have immediately struck you there were never ever [-----] reported infant Hep B cases in the US. The number of annual infant / perinatal cases before the first Hep B vaccine was developed in [----] and when Hep B was much more endemic was far far lower. That number really matters when you are talking about "catastrophic consequences"."
X Link 2025-09-20T01:18Z [---] followers, [---] engagements
"September 19th [----] $PRME continues to struggle to durably break out past the $5 range. As mentioned previously I think this has a decent chance of remaining true through the '26 INDs unless something happens with CGD. Though as also mentioned previously I think its true present value is a higher than what it currently is if one uses the $VERV buyout as a comp. $NTLA $PEPG and $NKTR all came back down following the run yesterday"
X Link 2025-09-20T01:27Z [---] followers, [---] engagements
"September 22nd [----] New high for the portfolio. Though still a lot of both risk and upside through the end of the year with particularly key data readouts for $PEPG $NTLA $RARE $NKTR $BBIO and $RNA beginning in the next week or so. $NTLA continues to push upwards $PEPG continues to operate at the upper end of the range it has been finding and remains my highest conviction position for greatest returns over the next few weeks"
X Link 2025-09-22T21:23Z [---] followers, [---] engagements
"September 23rd [----] Daily Update New ATH. Though as will be the case for awhile significant risk remains Big finish today for $PRME. I am curious if any of the price action was driven by shifting expectations around CGD based on the recent article and / or back channel chatter around accelerated approval. Time will tell whether its signal or noise"
X Link 2025-09-23T20:06Z [---] followers, [---] engagements
"Cue the shooters motives are unclear media cacophony quickly followed by shooter is MAGA because his third cousin once removed voted for Barry Goldwater FBI saying shooter's rounds had "anti-ICE messages." FBI saying shooter's rounds had "anti-ICE messages.""
X Link 2025-09-24T15:44Z [---] followers, [---] engagements
"@Cernovich Phonak is a good brand if he is going to an independent audiologist / ENT. However take a look at Costco. It's more cost effective and they carry the Jabra Enhance Pro [--] which is a really good option for patients with severe hearing loss. Know a few that had success with it"
X Link 2025-09-24T16:53Z [---] followers, [----] engagements
"@monaco_biotech Competing view: https://x.com/medstudentinvst/status/1970994289413898552 @Hall8Jack @blippd @scamsweeper Comparing dosing for $RNA or $ARWR to dosing for $PEPG has no meaning. $PEPG uses a cell-penetrating peptide conjugated to a phosphorodiamidate morpholino oligomer that sterically blocks the CUG repeats. $RNA and $ARWR use an siRNA that binds to the RISC and degrades the DMPK https://x.com/medstudentinvst/status/1970994289413898552 @Hall8Jack @blippd @scamsweeper Comparing dosing for $RNA or $ARWR to dosing for $PEPG has no meaning. $PEPG uses a cell-penetrating peptide"
X Link 2025-09-24T23:31Z [---] followers, [---] engagements
"Hypomagnesemia was the key potential safety issue observed in DMD51 not eGFR decline. However it was only Grade [--] / [--] hypomagnesemia was transient patients were asymptomatic kidneys showed no signs of structural damage and it resolved without intervention. In regard to eGFR yes you are right in that creatinine went up. However this was not considered reflective of a meaningful safety risk to the kidneys as cystatin C did not go up Cystatin C is a much better indicator of kidney function as creatine fluctuates for all kinds of reasons especially when dealing with peptides and muscle cells as"
X Link 2025-09-25T14:58Z [---] followers, [---] engagements
"September 25th [----] Daily Update New all time high driven by $PEPG result. That is one of the 2H [----] data readouts down. However a lot of readouts to go and significant risk remains. Particularly good to have the $PEPG result on a day when every other position in the portfolio (except for $QURE) was down"
X Link 2025-09-25T20:09Z [----] followers, [----] engagements
"September 26th [----] Daily Update End of week. $PEPG was obviously the highlight. This remains a long-term hold for me"
X Link 2025-09-26T20:03Z [----] followers, [----] engagements
"RA Capital effectively doubled its share count in $PEPG during $PEPG's most recent offering. As I have said elsewhere this company remains high risk given it has only shown Phase [--] data. However it cannot be stressed enough how much upside continues to exist here. DM1 represents at least [-----] patients in the United States with the majority being adult onset. If functional outcomes mirror the unprecedented improvement in splicing $PEPG will almost certainly dominate that market. Given ASO's are usually priced at least a couple hundred thousand dollar years annually that would translate into"
X Link 2025-09-30T23:51Z [----] followers, 12.1K engagements
"$CRSP put out a press release today about its new polymerase based editing technology and claimed the following: "SyNTase editors represent a significant advance over currently described prime editing systems by combining compact Cas9 proteins with a novel class of engineered polymerases. Together these components enable gene editing with greater efficiency and precision specificity while also supporting scalable manufacturing." My current sense this claim of superiority over prime editors is empty marketing fluff and is not supported by existing evidence. $PRME already appears to have"
X Link 2025-10-01T19:54Z [----] followers, [----] engagements
"October 1st [----] Daily Update. Fortunate to have the all-time high run continue. Though as said many times significant risk remains and things could easily reverse if I get a few things wrong this fall or biotech sentiment shifts back towards the negative. Interesting news from $CRSP today. I have posted my preliminary thoughts pending the more detailed data release in October in a separate post. $NTLA with a good gain today. We'll see if it holds. In truth day-to-day price action is rather unimportant relative to the data readouts this fall"
X Link 2025-10-01T20:06Z [----] followers, [---] engagements
"Im not up to speed on the patent law here. Do you know if SyNTase clearly infringes on prime-editing IP I asked Grok and Grok said its unlikely. That was rather shocking to me as its the most obvious way to copycat prime editing.just replace the RNA template and reverse transcriptase with a DNA template and a polymerase"
X Link 2025-10-01T20:30Z [---] followers, [---] engagements
"@tnguy093 Thanks dude. Well see if it holds"
X Link 2025-10-01T20:31Z [---] followers, [--] engagements
"@zhaoweiasu Awesome thanks. Very helpful. I would have been shocked if Prime did not have legal protection against a move like this. Curious if $CRSP will try to claim differentiation nonetheless based on the phrasing of their press release"
X Link 2025-10-01T20:55Z [---] followers, [---] engagements
"Cool thanks. Ill check out those post and will read their DNA polymerase patents in full this weekend. However based on my reading thus far I do think that $CRSP will almost certainly try to claim differentiation from $PRME on the basis of using synthetic nucleotide polymerases. Obviously intellectually and ethically SyNTase is a complete knock-off of $PRME editing. Anyone whos taken Biology [---] would see in a half a second that a DNA polymerase and DNA template will accomplish the exact same thing as an RNA template and reverse transcriptase the first time they read about prime editing."
X Link 2025-10-02T00:53Z [---] followers, [---] engagements
"As per my earlier post on SyNTase I continue to believe that $PRME possesses the leading gene editor technology and $CRSP s SyNTase is a prime editor knock-off that lacks competitive differentiation in specificity has shown no meaningful evidence of competitive differentiation in efficiency and is significantly behind $PRME in development. As always capturing even a few of the thousands of monogenic diseases would drive massive value appreciation for $PRME. However I also think it is likely that $CRSP will aggressively defend SyNTase in court as proprietary and novel in virtue of the fact it"
X Link 2025-10-02T01:55Z [----] followers, [----] engagements
"My man. I assure you I was not attacking you. I think we both agree that SyNTase is an obvious knock-off of prime editors and David Liu / Prime should have the right to this IP from an ethical / intellectual standpoint. I also think we both agree that $PRME retains a dominant lead in prime editing and nothing about SyNTase appears to change that. Im certainly not planning to decrease my position in response to this. Where it seems we differ is I think its highly likely that $CRSP believes they have found a possible legal path around $PRME s patents based on characterizing SyNTase as a"
X Link 2025-10-02T02:35Z [---] followers, [---] engagements
"I do appreciate the post dude. Its great stuff. However I suspect what $CRSP has done is altered the nucleotides in its templates with a few atoms that dont appear as of yet to do a ton functionally (though $CRSP will claim they drive differentiated function) CRISPR is going to subsequently claim that their polymerase engineered to transcribe those templates is a synthetic nucleotide polymerase and therefore outside the purview of $PRMEs DNA / RNA polymerase patents highlighted in your posts and elsewhere and therein $CRSP thinks they have a shot to win a legal fight even though SyNTase is"
X Link 2025-10-02T02:49Z [---] followers, [---] engagements
"I think youre missing my point here dude because Im not really tracking why you keep saying relax. First yes I know it was a press release. A central theme of my posts has been SyNTases differentiated capabilities are very likely overstated in the press release. It was the entire point of the very first post I made about SyNTase. In fact I think the overstatement of capabilities is likely important to $CRSPs legal argument. Second it seems you may be confusing my analysis of CRISPRs likely legal strategy based on the language they used in their press release with some kind of reactionary and"
X Link 2025-10-02T03:35Z [---] followers, [---] engagements
"This discussion is getting repetitive. However as said previously my point is that CRISPR almost certainly believes there is a legal defense that might work here as evidenced by the language of its press release and the fact that it would be grossly incompetent to pursue this without counsel providing a view on legal defensibility. Are you really disagreeing with this If $PRME ends up having to sue to enforce its patent that will cause a headache for them even if they prove victorious. Are you really disagreeing with that"
X Link 2025-10-02T03:51Z [---] followers, [---] engagements
"Hahahahaha okay Wei. Ive been patient with you. Now its clear youre an insecure asshole a mid wit and a waste of my time. Kindly fuck off. Im talking about CRISPR today because its relevant today. Are you slow And yes I know all about Tessera Tome (yes I know theyre winding down) Arbor Metagenomi and the rest. Knowing that is really not a badge of intellectual capability I promise. You can keep fantasizing I am saying $PRME is screwed (lol) so you have something to push back on. That doesnt change the reality that I have consistently stated I believe Prime remains the leading gene editor"
X Link 2025-10-02T04:38Z [---] followers, [---] engagements
"This is a phenomenal article. Kudos to the author. It also shows exactly why $CRSP will almost certainly aggressively make the argument I outlined yesterday to defend SyNTase from legal challenge and why they will lean heavily on their so called proprietary AI-guided structural modeling and large scale screening methods and their use of a synthetic nucleotide template to support the claim that synthetic nucleotide polymerases are novel and represent a significant advance as they interact with non-technical judges / juries. The Amgen case this author describes and Amgens failure to patent"
X Link 2025-10-02T14:06Z [----] followers, [----] engagements
"October 2nd [----] Daily Update Well needless to say $PRME and $NTLAand $CRSP had a day. $PEPG a nice rise as well and $PCVX continues to show some strength I do feel the need to always caveat that this continues to be a high risk portfolio and things could turn quickly. However pleased with the results thus far"
X Link 2025-10-02T20:08Z [----] followers, [----] engagements
"It looks like Viking also increased its position in $PEPG from [-------] at end of Q2 [----] to [-------] shares. Filed a 13G today"
X Link 2025-10-03T22:50Z [----] followers, [----] engagements
"@hannibalspeaks Yes exactly. I am George Washington's only living heir"
X Link 2025-10-03T23:03Z [---] followers, [--] engagements
"October 6th [----] Daily Update Reposting as the $ABVX close price was not updated"
X Link 2025-10-06T20:36Z [---] followers, [---] engagements
"@GeneInvesting Give it a rest Tony. This brand of insecure makes me want to dump every share of $NTLA I own. The stock could go to $200 and your returns still wouldn't come close to his"
X Link 2025-10-07T21:13Z [----] followers, [----] engagements
"Today is a good example of the benefits of the approach I outlined last night. I have effectively no insight into what non-event driven pricing in a biotech stock will do day to day. I could just as easily have seen $PRME and $NTLA being down 12% today rather than up 12% and 28% (for now) ______ As a small aside I do have a theory that biotech stocks especially over the last few years are an ideal sector for quant funds because of (a) the volatility and (b) there are likely extended timeframes where the drivers of intraday pricing are far more statistically predictable given the profile of"
X Link 2025-10-08T18:20Z [----] followers, [---] engagements
"Closed out the $RNA position today for effectively no gain given I think there are higher return opportunities in biotech. Put half into $QURE. The remaining half is in cash and will look to deploy in the future"
X Link 2025-10-08T18:25Z [----] followers, [---] engagements
"October 8th [----] Daily Update All-time high. $NTLA and $PRME with big days. Though I would not be surprised to see continued volatility and a retraction. The gains for $NTLA are really little more than just noise until the data readouts in a few weeks. As mentioned in a previous post I exited $RNA at an effectively flat valuation. This is largely a function of believing there are biotech companies with similar risk profiles that have a higher upside rather than a negative view of $RNA. Certainly could be bought out and there is likely upside there. I initially entered $RNA as a relatively"
X Link 2025-10-08T20:16Z [---] followers, [---] engagements
"$ABVX didn't update again and just realized so here is the corrected daily update for $ABVX's actual close price. No change to headline total return"
X Link 2025-10-08T20:21Z [---] followers, [---] engagements
"October 9th [----] Daily Update All-Time High. Still super risky portfolio given it's biotech. $NKTR and $NTLA probably the companies with the biggest potential catalysts over the next few weeks"
X Link 2025-10-09T20:35Z [----] followers, [----] engagements
"Mostly just wanted to put the cash towards $NKTR. My view on $VKTX continue to be it likely has a market and upside if it maintains headline efficacy lead in the GLP-1 / GIP agonist class regardless of any side-effect incidence matched comparisons though competition from big players is intense and CMC remains important. I think it's likely continue to show in upcoming data readouts"
X Link 2025-10-15T14:53Z [---] followers, [---] engagements
"@AdamBLiv Alternatively you could just buy an asset that has a 127% CAGR. Neither a 401k nor bitcoin necessary. I am the quant now"
X Link 2025-10-15T18:13Z [---] followers, [--] engagements
"Alternatively you could just buy an asset that has a 127% CAGR. Neither a 401k nor bitcoin necessary. I am the quant now. People dont like hearing this but putting money your 401(k) just to get the employer match is stupid. Most people dont realize their 401(k) match is just a leash. Free money sounds nice until you realize its denominated in melting ice cubes and you cant touch it for [--] People dont like hearing this but putting money your 401(k) just to get the employer match is stupid. Most people dont realize their 401(k) match is just a leash. Free money sounds nice until you realize its"
X Link 2025-10-15T18:14Z [---] followers, [---] engagements
"I do think given the $VERV $1Bn buyout the investment is derisked somewhat regardless of the day to day price movement in a fire sale scenario unless there's a major prime editing platform safety event It's interesting if you go back you'll see $ALNY had its issues and its volatility for a long time. So a sign of hope there https://twitter.com/i/web/status/1983989106851115029 https://twitter.com/i/web/status/1983989106851115029"
X Link 2025-10-30T20:07Z [----] followers, [---] engagements
"Come on man. Thats not good faith. You really believe what I wrote means Im comping $PEPG today to $ALNY or $BBIO Might as well say Im comping $PEPG to $PFE while youre at it. It should be abundantly clear I am drawing the comparison between $PEPG s EDODM1 and $BBIO and $ALNY s ATTR-CM drugs to assess the ability of an approved post-phase III best-in-class drug in DM1 if EDODM1 makes it that far to gain 10% market share in the US market which was the point you questioned. On the prevalence point using that worldwide number is purposefully obfuscatory when I was citing US numbers for DM1. We"
X Link 2026-01-04T02:31Z [----] followers, [---] engagements
"The content of the article itself is fine. Just says $ABVX explicitly declined to comment on any M&A / auction processes and that Marc confirmed a formal review by the French government can't occur until there is a publicly announced offer which speaks directly to the statement by the French government that there had been no discussions with $LLY. It's the headline Reuters chose that's the issue as it erroneously suggests Marc denied the existence of any auction process / M&A discussions on the basis of Marc's comment that $ABVX can't worry about market "noise" and they just need to execute"
X Link 2026-01-20T18:03Z [----] followers, [---] engagements
"$PEPG position initiated this morning based on what I see as great reward to risk ratio leading into critical October [----] data readout. If drug demonstrates efficacy think at least 4x return possible off that data readout alone. Long term return if successful much greater. Key is to keep position size small enough for portfolio to absorb losses if fails while still having enough size to capture upside at the portfolio level. If drug fails downside perhaps protected by negative EV and non-zero likelihood RA Capital / Viking Global / Point72 successfully push $PEPG to return cash to"
X Link 2025-08-14T16:54Z [----] followers, 16.8K engagements
"$PCVX Believe it is likely the NIH statement referenced below that has driven the slight uplift seen over the past week. Below can see Prevnar ( $PCVX 's key competitor) numbers revenue numbers. $PCVX market cap is currently $4.25Bn. Taking 30% of this market share would yield $1.9Bn in revenue and $9.5Bn EV off 5x revenue multiple. 50% of this market share would yield $3.2Bn in revenue and $16.0Bn EV off 5x revenue multiple. 75% would yield $4.0Bn revenue and $20Bn EV off 5x revenue multiple. As stated previously historical norm for commercial stage biotechs is 5.5x - 7.5x revenue multiple."
X Link 2025-08-16T04:17Z [---] followers, [---] engagements
"August 20th [----] Used proceeds from selling half of Alnylam position (+94.1%) yesterday into $NKTR and $ABVX (about half into each). $VKTK thoughts from yesterday - This trade really emphasized to me the importance of staying tight on three things: [--]. Entry price - A good business is a bad investment if it is bought at too high a price. A mediocre business can be a good investment if bought at a cheap enough price. Of course one can take that principle too far overly optimize for entry price and miss out on an opportunity because the stock runs. In such cases if the expected return is"
X Link 2025-08-20T21:13Z [---] followers, [----] engagements
"August 22nd [----] $PRME getting a nice little rip at the end of the week thanks to Jerome. $WVE has had a nice run recently. The limited reaction from $PCVX $NTLA and $RARE (fall / winter readout stocks) raises the probability of muted upside until their fall readouts hit. $ABVX continuing to inch up"
X Link 2025-08-22T21:06Z [----] followers, [----] engagements
"Another thought from the drafts: There are issues with mice data provided by $ARWR. First competing mice data appears to show the lipolysis driven by INHBE / ALK7 silencing ( which $WVE / $ARWR would acknowledge is the purpose of the therapy) does lead to lipotoxicity and signs of insulin resistance like hepatic steatosis within certain metabolic contexts. I've put one of the studies (there are multiple) showing this below. Additionally take a look at the attached screenshot of $ARWR 's glucose / insulin sensitivity data. The tests they are running are probably not sensitive enough to detect"
X Link 2025-09-11T16:16Z [---] followers, [----] engagements
"$QURE news is good news for patients more bad news for $WVE $WVE: [--]. RNA editing platform has demonstrated inferior efficacy relative to DNA editing / base editing thus far [--]. Obesity / INHBE silencer may drive increased insulin resistance in patients who are already obese as laid out in previous posts. Competition with $ARWR at baseline [--]. One and done Huntington Disease therapy from $QURE sets a high bar for efficacy $WVE will need to match or exceed $QURE is one and done vs $WVE s continuous dosing regimen and $QURE is in more advanced stages than $WVE [--]. DMD therapy (exon 53) only"
X Link 2025-09-24T14:15Z [---] followers, [---] engagements
"$PEPG Well there you have it. It shall indeed likely be the highest gaining position over this short-term time frame. It was just such an unbelievable opportunity to be able to buy it in the low $1's. For reference $PEPG's key competitors (which it appears to blow out of the water) are multi-billion dollar companies ( $RNA and $DYNE). https://twitter.com/i/web/status/1970962013049573862 https://twitter.com/i/web/status/1970962013049573862"
X Link 2025-09-24T21:22Z [----] followers, 12.6K engagements
"September 30th [----] Daily Update This can't continue forever. but another all-time high today largely on the strength of $PRME. Good to see $PCVX back in the green too after announcing a $1Bn manufacturing deal with Thermo Fisher. My sense is $PCVX will be a beneficiary of a couple things: [--]. The market's realization that NIH / FDA isn't anti-vaccine it's anti-stupid vaccine. Or said more academically it's anti-vaccine for vaccines that have an unclear risk-reward tradeoff. In the United States pneumococcal pneumonia is estimated to result in approximately [------] hospitalizations each year"
X Link 2025-09-30T23:52Z [----] followers, [----] engagements
"Id say $QURE $PCVX $RARE $NKTR $ABVX are high growth potential with somewhat more limited risk than the others. However biotech is always a very very high risk sector and I cant predict any outcome in biotech with certainty so if youre going invest in those please only do so if youre fully comfortable taking big losses in the positions. Obviously none of this is financial advice. If the risk parameters were widened just a bit I would also throw $NTLA in there as good risk / reward. Quite high upside and key data readouts upcoming in the next few weeks with good visibility into what the"
X Link 2025-10-04T00:26Z [----] followers, [----] engagements
"Hypothesis for a contributor to $PEPG s differentiated splicing improvement in DM1. $RNA and $DYN use DMPK1 RNA silencers which degrade and destroy the DMPK mRNA. In contrast $PEPG s ASO binds to the CUG repeats and sterically blocks the hairpin loop formation allowing the DMPK mRNA transcript to be translated normally. Most biological systems have negative feedback loops. So an increase in expression production or activity or a molecule will trigger inhibition of the expression production or activity of that molecule. Given $PEPG allows the DMPK mRNA to operate normally and produce the DMPK"
X Link 2025-10-04T08:55Z [----] followers, [----] engagements
"They have a very different risk / reward profile. $MREO does not have an existing commercial business generating hundreds of millions of dollars. If setrusumab fails $MREO's stock price is therefore likely to fall much more than $RARE's. We saw that after setrusumab missed at IA2. Alvelestat almost certainly will be non-competitive with $BEAM $PRIME 's and $WVE 's AATD therapies"
X Link 2025-10-05T18:53Z [---] followers, [---] engagements
"The failure of $SKYE s therapy is notable today in the context of INHBE / ALK7. The primary lipolysis enhancing effect of INHBE / ALK7 silencers appears to be driven by an increase in intracellular cAMP and PKA via inhibition of pathways that decrease cAMP and PKA concentrations. As noted elsewhere PKA drives activation of lipolysis enzymes (e.g. HSL and ATGL via perilipin). $SKYE s therapys primary downstream effect is also enhanced lipolysis through upregulation of cAMP and PKA concentrations via inhibition of pathways that decrease cAMP / PKA concentrations. It just works the CB1 receptor"
X Link 2025-10-06T12:19Z [---] followers, [---] engagements
"Right. OI represents the predominant driver of $RARE s upside. However the baseline commercial business of $RARE is probably worth around $28 per share. That provides substantial downside protection if setrusumab fails at the Final Analysis. In such a scenario $RARE will probably trade no lower than mid-teens immediately following the news and still has a reasonable chance to recover to those high twenties levels and higher if Angelman proves to be a commercially successful therapy. In contrast If setrusumab fails $MREO will be wiped out and thats particularly true if Alvelestat is considered"
X Link 2025-10-06T16:59Z [---] followers, [---] engagements
"Im not really sure what there is to debate here. Yes setrusumab has a reasonable probability for success hence my investment in $RARE. However it quite obviously does not have a 100% chance of success. Consequently $MREO carries more risk into the FA with $MREO s downside case being a complete decimation of the stock and $RARE s downside case ranging from mid teens to effectively flat. Do you disagree with that As I have said I dont want $MREO s binary risk exposure when $RARE has likely 2x upside with a baseline commercial business generating hundreds of millions of dollars in revenue. If"
X Link 2025-10-06T18:01Z [---] followers, [---] engagements
"Im not really sure what there is to debate here. Yes setrusumab has a reasonable probability for success hence my investment in $RARE. However it quite obviously does not have a 100% chance of success. Consequently $MREO carries more risk into the FA with $MREO s downside case being a complete decimation of the stock and $RARE s downside case ranging from mid teens to effectively flat. Do you disagree with that As I have said I dont want $MREO s binary risk exposure when $RARE has likely 2x upside with a baseline commercial business generating hundreds of millions of dollars in revenue. If"
X Link 2025-10-06T18:02Z [---] followers, [---] engagements
"$ALNY has returned 76.5x since inception. $10000 invested at $6 would be $766500 today. $50000 would be $3.83M today. $100000 would be $7.66M The picture attached is $ALNY 's stock chart. This represents the argument for why to stay long in the face of volatility when invested in early stage biotechnology companies which you believe have a very good chance of seeing enormous value accretion over the long-term based on TAM competitive differentiation current valuation etc. $ALNY 's share price saw significant volatility over the years. It collapsed soared plateaued and everything in between."
X Link 2025-10-07T22:12Z [----] followers, [----] engagements
"October 10th [----] Daily Update I allocated the remaining $RNA cash to $JANX today. If $NKTR continues to move down I will likely exit the tiny $BEAM and $VKTX holdings and put those towards $NKTR. Portfolio as a whole drew back about 7.3% today largely driven by the top [--] positions in the portfolio ( $PRME $NTLA $PEPG). I expect $PRME and $PEPG to continue to experience significant volatility over the short to medium term and consider these price movements irrelevant unless my long-term thesis on the business changes or new data emerges. Though it means the portfolio as a whole is likely to"
X Link 2025-10-10T23:25Z [----] followers, [----] engagements
"Just closed small remaining positions in $VKTX and $BEAM. Proceeds went into $NKTR. Portfolio weights will be updated in daily update tonight"
X Link 2025-10-15T14:45Z [---] followers, [---] engagements
"$PCVX continues to inch-up. They still have data to show of course. However in my personal view it was an absolute steal a steal to be able to get this in the 30's for a company as large it is with such a strong a cash position. Surprised it took as long as it did to see some of the sector gains biotech in general has seen especially after NIH made its vaccine position abundantly clear. $RARE is also inching up. Setrusumab is still an open question and is by no means guaranteed. However from a risk-reward perspective given downside is protected by the commercial business and has two other"
X Link 2025-10-15T15:44Z [----] followers, [---] engagements
"Lorenzo "Botswana" (who has $1Bn of AUM a PhD [--] other acronyms and clips $20M of annual management fees given he obviously is a [--] and [--] guy minimum) shorted $SOC at $30 as announced after the fact. Word is he was also long $SPRB as announced the day after it increased [--] trillion percent. Are there any investor materials available for a highly interested prospective LP such as myself I shorted $SOC at $30 because the dumbest motherfuckers Ive ever seen own that stock. They do not know the method. Elevate. 💯 I shorted $SOC at $30 because the dumbest motherfuckers Ive ever seen own that"
X Link 2025-10-15T16:15Z [----] followers, [----] engagements
"Early Daily Update for posterity as I might never see this again and this whole thing could easily collapse tomorrow. The current portfolio is all green. The big glaring red mark is that dumb throwaway $MGX position I exited. The intrusive thoughts to re-enter and try and ride it until I erase that -13.5% are flying simply fast and furious rn https://twitter.com/i/web/status/1978540347014946998 https://twitter.com/i/web/status/1978540347014946998"
X Link 2025-10-15T19:15Z [----] followers, [---] engagements
"Wednesday October 15th [----] Daily Update All-time high. Though always needs to be said this is a high risk portfolio and there are a number of upcoming events that could turn the ship right around. As mentioned closed the very small $BEAM and $VKTX positions today and put virtually all remaining cash into $NKTR"
X Link 2025-10-15T20:08Z [---] followers, [----] engagements
"October 16th [----] Daily Update Markets go weeeeeeeeeeeeeeee portfolio comes back into the 70s after all but $RARE is red today. $RARE (and $PCVX to an extent) showed the most resilience. Again these are names that I think are likely to have high institutional interest and have not experienced the same run as some of the others. So not entirely surprised to see that"
X Link 2025-10-16T20:16Z [----] followers, [---] engagements
"Totally agree DM1 is a huge market and $pepg very interesting. Re $WVE and INHBE may want to check out the attached post. I think there is a fairly high likelihood that $WVEs approach of further increasing adipocyte / hepatocyte lipolysis through INHBE silencing will worsen insulin sensitivity in patients who are already obese and almost certainly already have excess lipolysis as the baseline excess lipolysis is a major causal factor in insulin resistance / diabetes. I think there is a core difference in molecular profile between patients who have LoF INHBE gene variants and therein"
X Link 2025-10-17T22:00Z [----] followers, [----] engagements
"Sorry I am just seeing this. Thank you I think it has a better chance in a maintenance role once adipocyte lipolysis has normalized somewhat post GLP-1. However a key issue it will need to overcome is that obesity tends to leave a lot of durable molecular signatures that promote weight gain even after an obese patient has lost weight that are not present in the never obese patients who experience the LoF INHBE mutations. Thats the main reason keeping weight off is so hard for the formerly obese. I think durable molecular signatures that have effects on appetite drive play the most biggest"
X Link 2025-10-21T16:59Z [---] followers, [--] engagements
"As I said at the time of the press release it was blindingly obvious $CRSP was going to aggressively argue SyNTase was a substantively different technology than prime editors and outside the scope of $PRME's patents regardless of whether that position is ethical or intellectually honest. As I also said at the time this may prove to be a headache for $PRME even if it wins out or gets licensing fees in the end. Legal outcomes are impossible to predict and legal processes are expensive. $CRSP can bear legal costs a lot more comfortably than $PRME can. $CRSP 's intentions and their likely legal"
X Link 2025-10-22T15:49Z [----] followers, [----] engagements
"For headline clarity (see back half of the post) I believe $PRME remains the clear leader in gene editing. I think it remains a phenomenal venture style investment over the long-term for the [--] reasons highlighted derisked barring a black swan safety event based on the $VERV buyout at the depths of the bear market and remains my largest position. I have not sold a share and nothing about this situation causes me to change that weighting. This is commentary about CRISPRs intentions the legal situation and possible impacts on short to medium term cash and operational dynamics for $PRME which may"
X Link 2025-10-22T17:47Z [---] followers, [---] engagements
"October 22nd [----] Daily Update Those gene editing stocks sure can be volatile. Large pullback the last coupe of days. Total return actually dipped into the high 60s at one point. If $NTLA $RARE and $PCVX can show good data and $NKTR gets some good news in AA one small upside of that will be a degree of smoothing out in the some of the headline volatility somewhat that is an inherent part of owning a very large position in $PRME. Though in truth the volatility doesn't really matter only the end outcome. Very curious to see what $NKTR has to say about the asthma sub-group in its Nov update"
X Link 2025-10-22T20:11Z [----] followers, [---] engagements
"October 23rd [----] Daily Update All eyes on early November👀( $NTLA $NKTR)"
X Link 2025-10-23T20:03Z [----] followers, [----] engagements
"October 24th [----] Daily Update The positions are set and the Q4 data gauntlet begins. The portfolio could look very different by the end of the next two months for better or worse. $BBIO will announce the results of its LGMD2i/r9 therapy on Monday morning. This will have a relatively small impact on the portfolio either way due to the position size and its small relative contribution to $BBIO's valuation. However early November will bring higher impact data readouts. The most notable is $NTLA"
X Link 2025-10-24T23:20Z [---] followers, [---] engagements
"Bad news from $NTLA. This will have significant price impact and those effects will ripple across the gene editing sector. As it stands my position is at a 1.8% loss. So where do we go from here Grade [--] elevations have happened before in Q2 '22 (technically I don't believe it was specified as Grade [--] but I am pretty confident it was) and May [----]. Thus Grade [--] elevations are a known phenomenon and at this point have occurred enough that it appears to be an inherent risk of the therapy. If this Grade [--] elevation is a mere plasma elevation with have no clinical symptoms or structural liver"
X Link 2025-10-27T12:55Z [----] followers, [----] engagements
"I have closed my $NTLA position for a small gain (will be shown in portfolio update tonight). Below I will break-down my rationale for exiting and detail a hypothesis for what may have happened My investment in $NTLA was largely based on the following: [--]. $NTLA demonstrated the ability to stop (not just slow) functional decline over the timeframe studied in an unprecedented percentage of patients with transthyretin amyloidosis a fatal disease that leads to heart failure polyneuropathy and a whole host of downstream consequences not well described in the medical literature or recognized"
X Link 2025-10-27T15:34Z [----] followers, [----] engagements
"If this safety event does not prove to be what it initially seems or there is a clear way to identify patients at risk for the disease I will revisit this decision. However for now I am out"
X Link 2025-10-27T15:34Z [---] followers, [---] engagements
"Finally a hypothesis for what happened. I posted elsewhere (will try to find it today) that one of the under appreciated risks of the gene silencer companies (first-gen CRISPR / $NTLA / $CRSP) is that the way they are intended to work is through the stochastic alteration of a targeted genes genetic sequence through indels which renders it unable to make a functional mRNA transcript and leads to the natural degradation by the cell of that transcript however it is well described that certain mutated mRNA transcripts can lead to toxic effects and its possible the random deletion or insertion of"
X Link 2025-10-27T15:34Z [----] followers, [---] engagements
"It remains to be seen what emerges regarding the underlying cause. I think one hypothesis that has merit (but is simply a hypothesis) is the indels introduced by $NTLA's cas9 at the site of the DSB create a new genetic sequence which codes for a toxic mRNA perhaps limited to a small subset of patients because a second baseline mutation is required for toxicity (the TTR protein has hundreds of natural occurring variants). Mutations causing toxic mRNA is a well-described phenomenon in other contexts If this hypothesis were to be correct it would have no effect on $PRME as prime editors are"
X Link 2025-10-27T18:34Z [---] followers, [---] engagements
"October 27th [----] Daily Update I posted my thoughts on the disappointing $NTLA news my original thesis why that thesis changed post safety event and a hypothesis for what happened mechanistically earlier today. That is a more complete breakdown. However at the portfolio level wrt $NTLA today was a good example of entry price playing an important role in the risk-reward profile of an investment. Despite a potential program threatening safety event was able to close the position with a 2.2% gain and the portfolio as a whole was only down about 5% from Friday. Believe upside was worth such a"
X Link 2025-10-27T20:42Z [----] followers, [----] engagements
"The short answer is I do with the primary reasons being: [--]. Large market size of AD [--]. Unique MoA [--]. Strong efficacy to date [--]. Very favorable side effect profile (personally I would choose ISR risk profile over the others) [--]. Likelihood of t-reg selective IL-2R agonist success in other indications (alopecia areata in the nearer-term and potentially others beyond) One of the elements I find most exciting about t-regs is they are biological entities designed to modulate their effector functions in response to the surrounding immune environment in highly complex ways. Thus while IL-2R agonism"
X Link 2025-10-27T21:17Z [---] followers, [---] engagements
"The first liver enzyme elevation was in of itself irrelevant nex-zs superior efficacy to existing alternatives as it was transient and had no clinical consequence. If this event fit the May [----] event profile I would not have sold a share of my position. Vutri does not do the same job as nex -z and this is well established in the clinical data. This should not be in question and if it is it just means you are neither familiar with the data nor the disease. Nex-z stabilizes functional decline for a significant percentage of patients. Vutrisiran does not. I assure you if a family member was"
X Link 2025-10-27T21:36Z [---] followers, [--] engagements
"I think $NTLA bears and bulls are talking past each other right now and many on both sides are missing a key piece of the equation - nucresiran. Bulls say $NTLA's nex-z results in a significantly lower percentage of patients showing disease progression relative to $BBIO's acoramidis and $ALNY 's vutrisiran. This is empirically true and it really does matter. Progression of heart-failure is tremendously serious and it is critically important to patient outcomes to fully arrest it as early as possible to prevent the panoply of downstream consequences like cognitive decline kidney dysfunction"
X Link 2025-10-28T10:53Z [----] followers, [----] engagements
"October 28th [----] Daily Update Position in $RCKT initiated today. Very high-risk. Quick and dirty thesis posted earlier today. Three shots on goal to see returns. Position size key so failure across all three will not materially impact portfolio to the downside but success in at least one will. $BBIO announcing data on ADH1 tomorrow. Very nice suite of therapies at this point. $QURE continues to move in a positive direction $PEPG back down. Honestly I had a tough time seeing that jump post $RNA acquisition being durable. It seems to me much too abstract in its relevance to $PEPG to support a"
X Link 2025-10-28T20:56Z [----] followers, [---] engagements
"Whoops typo in the first sentence - PKP2-ACM as seen below. Quick and dirty for real"
X Link 2025-10-28T20:58Z [---] followers, [---] engagements
"I certainly thought so before this safety event. If the only notable side effect was rare and transient liver enzyme elevations that resolved without medical intervention i think there was a good probability that nex-z would have found success and seen large returns relative to its recent lows. I personally would have had a tough time prescribing tafa / acora / vutri over it given nex-z gives a patient a meaningfully better chance at seeing no disease / heart failure progression (as measured by pro-ntBNP / functional tests) at least over the timeframe studied and that really matters"
X Link 2025-10-29T01:46Z [---] followers, [--] engagements
"Really fantastic results so far this week from $BBIO through and through. A very well-run company that seems to do things the right way. Really appreciated the way they provided a free lifetime supply of acoramidis to patients. Makes them easy to root for. I love the simplicity of encaleret. It's an oral drug that directly targets the key underlying pathophysiology of ADH1 and its MoA means it's unlikely to significantly perturb endogenous pathways whilst doing so. My eyes now turn to what is going to happen with tafamidis' patent protection. If Pfizer is able maintain tadamidis patent"
X Link 2025-10-29T12:11Z [---] followers, [---] engagements
"October 29th [----] Daily Update Pullback continues in the portfolio though feel pretty good about the positions as they stand. $PRME continuing to trend down post $NTLA news is the biggest contributor to the ongoing pullback. For a whole host of reasons I posted about yesterday I do not believe there is a read through from $NTLA to $PRME. One of the reasons I listed and haven't seen discussed much (most of the focus is on the LNP / the cas9 mRNA / protein immunogenicity) is it is possible $NTLA's issues flow from cas9 induced indels which are a design feature of first-gen CRISPR and not prime"
X Link 2025-10-29T21:31Z [---] followers, [----] engagements
"Just FYI $ALNY 's nucresiran (only 1-2 years behind nex-z) will almost certainly match nex-z's efficacy based on depth and speed of TTR knockdown. I would have a very hard time feeling comfortable prescribing nex-z if I had an equally effective drug that didn't carry the risk of a DILI in nucresiran (2x per year dosing). $NTLA's valuation depends on long-term nex-z prescriptions. Something to consider as I think $NTLA is at risk in a way it has never been before"
X Link 2025-10-29T22:32Z [---] followers, [---] engagements
"An additional point in support is $ALNY 's nucresiran will almost certainly show the same efficacy as nex-z based on rapidity and depth of TTR knockdown with no DILIs. So nex-z's point of differentiation in having a clinically meaningful efficacy advantage over acora / tafa / $ALNY's vutri gets obviated as soon as nucresiran comes to market 1-2 years behind and there then isn't even an efficacy argument to support a prescription of nex-z"
X Link 2025-10-30T17:07Z [---] followers, [---] engagements
"I know man. it's tough to see. I expect that this will be a bumpy ride for awhile. Deserved or not I unfortunately think $NTLA was acting as a bellwether / canary in the coal mine of sorts for in vivo gene editing in the markets' eyes and I expect $NTLA's very real issues with DILI to ripple through the sentiment around the rest of the sector for awhile. $PRME will no longer get the market confidence boost from an in vivo editor making in to market without notable issues. I continue to believe there is a good chance that the DILI is a function of the stochastic indels first-gen CRISPR is"
X Link 2025-10-30T19:51Z [---] followers, [---] engagements
"Also just added to $ABVX. Basically all time highs but perhaps the waters are swirling and want to favor being in market with capital during a bull run rather than out"
X Link 2025-10-30T19:55Z [----] followers, [----] engagements
"October 30th [----] Daily Update Explained reasoning behind changes made today in previous posts. [--]. Closed $CRSP [--]. Opened $PASG [--]. Opened another $ABVX position"
X Link 2025-10-30T20:29Z [----] followers, [----] engagements
"The Lilly trial is still enrolling unless youve seen something Ive missed The death in their trial was not considered related to the therapy and the DRG issues have not appeared in the Dose [--] (high dose cohort w/ supraphysiplogic progranulin expression) for $PASG. Dose [--] is actually a 50% lower dose"
X Link 2025-10-31T17:17Z [----] followers, [---] engagements
"Reposting an excerpt of a hypothesis I put out a few days ago regarding the underlying cause of $NTLA s DILI. I could have sworn I also posted about this specific risk some months back though have not been able to find the post and it may have just been in my notes / thesis amidst possible risks. The hypothesis didnt drive returns as until the DILI it was theoretical to my knowledge. However it prevented me from losing money on the investment as that hypothesis along with the existence of nucresiran drove my $NTLA exit post DILI. It also shows why there is a low probability of a read through"
X Link 2025-10-31T23:53Z [---] followers, [---] engagements
"October 31st [----] Daily Update Added a little more to $PASG to close out the week"
X Link 2025-11-01T01:24Z [----] followers, [---] engagements
"@plainyogurt21 @aditharun_ Makes sense some of the side effects that emerged with $LLY s (e.g. DRG issues) havent been seen to date with $PASG s early data so perhaps will prove to be an additional point of differentiation. Though AAV always risky"
X Link 2025-11-01T17:14Z [----] followers, [---] engagements
"This looks suspiciously like an unattributed rip-off of the hypothesis I articulated across a number of earlier posts including in a comment on this guys own post stating his initial view was that transient LNP / cas9 immunogenicity was the cause of the DILI. If thats true ironic considering our only prior interaction was him taking issue w/ the clear reality that $CRSP will try to argue SyNTase was novel on the basis of non-PACE development and the use of synthetic nucleotides despite it being an obvious rip-off of prime editing. Wherever he got the neo-epitope idea from his proposal for an"
X Link 2025-11-03T01:32Z [---] followers, [----] engagements
"I do grasp what you are saying. I understand this is not for all patients. Indels are stochastic and only a couple out of hundreds had a clinically observable issue. It doesnt matter. If DILI is a risk doctors will go with nucresiran. It would be unethical to do otherwise assuming equal efficacy If you want to bank on HAE more power to you"
X Link 2025-11-03T02:53Z [---] followers, [---] engagements
"Agreed the cause is still unknown and any outcome is possible. However its actually far more complicated than just a simple sequencing of the TTR gene. You first have to take a liver biopsy to even attempt to sequence the TTR gene. Probably dont want to do that to a patient who just met Hys Law. Even if you could take a biopsy you have to keep the biopsy small and hope that sufficient liver cells captured in the biopsy have both been edited and been able to survive any immune attack apoptotic or toxic mechanism. If that doesnt happen the cells wont exist in the biopsy to be sequenced. Amongst"
X Link 2025-11-03T08:37Z [---] followers, [---] engagements
"I have a feeling this $QURE issue may not last overly long You never know. However if there was ever a disease that warranted a right to try Huntingtons Disease is it and the President has consistently backed the right to try. I also dont think Trump will remain uninvolved if the HD community starts getting vocal (as they likely will)"
X Link 2025-11-03T13:22Z [----] followers, [----] engagements
"Okay this is just a musing and Im not saying this is the case. So dont go buying a bunch of $QURE on the basis of this However does the language of the $QURE press release not suggest its possible $QURE may be playing hardball with the FDA / Vinay here Perhaps the meeting with the FDA was more contentious than theyd like Vinay threw his weight around and theyre trying to leverage public pressure (as was seen with Elevidys DMD patients and Sarepta) to make sure they get the outcome they want Basically a jump the chain of command move to get to Marty / RFK or the President who all clearly"
X Link 2025-11-03T15:55Z [----] followers, [----] engagements
"Needless to say the $QURE news has read through to $PASG. Just so anyone who read my post about investing in it is also aware of this new risk / downside. I plan to continue to hold as I believe its FTD-GRN therapy's profile and current market cap continue to present impressive risk-reward regardless of the ultimate confirmatory trial structure / pathway. Recall that $LLY brough proclaim for $880M plus a CVR and $QURE is still worth $1.89Bn. I also think we're a long way from the conclusion of this $QURE story. However I would be remiss not to acknowledge this and the risk definitely just"
X Link 2025-11-03T16:25Z [----] followers, [----] engagements
"I also bought more today so do with that what you will"
X Link 2025-11-03T16:39Z [----] followers, [---] engagements
"@OppInv More $PASG :)"
X Link 2025-11-03T17:15Z [----] followers, [---] engagements
"The $QURE news is actually less of a read through to $PASG than I initially feared based on this. The FDA appears to be taking issue with a failure by $QURE to define the statistical analysis plan (SAP) before study initiation. While I do think HD patients deserve the right to try with AMT-130 as soon as feasible given the severity of the disease and the lack of therapeutic alternatives and $QURE seems to have a legitimate claim that the FDA is flip-flopping in its stance asking for SAP to be defined ahead of trial initiation is actually quite a reasonable stance in most situations otherwise"
X Link 2025-11-03T20:30Z [----] followers, [----] engagements
"November 3rd [----] Daily Update. Added a little bit of new capital into the portfolio to be able buy some more $PASG. Return today before capital addition was 52.5%. Return after new capital is 51.3% as of course the "starting capital" denominator went up and new capital has an effective return of 0%. The latter return is listed. I have posted my existing thoughts on the $QURE pullback today elsewhere. I plan to hold. The portfolio level thing I have to figure out post the $QURE news is whether this portfolio remains appropriately weighted from a risk perspective. I had marked $QURE as a"
X Link 2025-11-03T22:49Z [----] followers, [----] engagements
"Today is so exemplary of just how wild biotech can get. $QURE multi-billion dollar company and media darling down over 50% after basically everyone (few notable exceptions) thought it was an smooth road to BLA submission and approval $SRPT was worth over $15Bn in [----] now down in the [--] billion range Not for the faint of heart boys and girls"
X Link 2025-11-03T23:07Z [----] followers, [----] engagements
"Literally every investor in biotech at some point (probably will be me after $RARE tonight) The pre / post market FUCK is just so real ðŸ˜ðŸ˜”😂"
X Link 2025-11-04T15:12Z [----] followers, 196.3K engagements
"comedic gold @MartinShkreli"
X Link 2025-11-04T15:22Z [----] followers, [----] engagements
"All the idiots buying bitcoin fail to realize that $VKTX is the real store of value. Hashtag uncorrelated asset"
X Link 2025-11-04T15:53Z [---] followers, [---] engagements
"@grok @GeneInvesting Thank you Grok"
X Link 2025-11-04T17:04Z [---] followers, [--] engagements
"@GeneInvesting @grok Oh Tony youre adorable Here it comes. the all-mighty PATIENT SUBTYPPPINNGGGG I get more excited for the full thread by the minute"
X Link 2025-11-04T17:06Z [---] followers, [--] engagements
"Tony (fuming he doesnt know MHC and HLA are the same thing): I dont read your posts man Also Tony: Ive commented on your posts like [--] times in the last couple of days Did you rip-off the I dont read your posts line off from someone too Or did you ask Grok for ideas on how to deflect when your takes have been disproven Now talk to JLaw"
X Link 2025-11-04T19:01Z [---] followers, [--] engagements
"November 4th [----] Daily Update The beatings shall continue until morale improves. Biotech getting absolutely whacked today. Every position I hold was down except $PEPG (). which came in at a really good 0% return for the day. It sure would have been nice to start entering those $RCKT / $PASG positions a few days later.but c'est la vie. $RARE got whacked too after hours on revenue and EPS misses. So I except tomorrow could be bloody. However whilst it's not ideal to lose Crysvita revenue as part of the North American Royalties sale to OMERS they announced this evening it does allow $RARE to"
X Link 2025-11-04T23:57Z [----] followers, [---] engagements
"Good info. Thanks to the OP. My takeaways from this for a registrational study for $PASG s PBFT02 would be: [--]. Include a quantitative volumetric MRI analysis as an endpoint (MOST CRITICAL) [--]. Easier to run an RCT ( if FDA requires) with PBFT02 as the surgery is much less invasive than $QURE s. $PASG s is more like a lumbar puncture [--]. Extra critical to align with FDA (not that this appeared to help with $QURE) on endpoints in non-RCT setting as CDR-FTLD is more susceptible to test administrator subjectivity / bias (i.e. less objective measures) [--]. CDR-FTLD is less susceptible to practice"
X Link 2025-11-05T01:23Z [----] followers, [----] engagements
"I would really encourage retail investors to exercise caution listening to this guy's statements on $NTLA. He has every right to express himself. However you should know he is carrying a very large bag he has pretty consistently demonstrated he is not a good-faith actor in discussions on $NTLA - often failing to meet the baseline standard of accurately representing the substance of opposing views and is willing to use scientific concepts and terminology he appears not to fully understand to justify what is clearly a preordained conclusion that " it was the right move to put 100% of my money"
X Link 2025-11-05T20:28Z [----] followers, 14.7K engagements
"One last thought that is actually to $NTLA 's benefit though definitely wouldn't hang your hat on this. Just one of the first approaches I'd take if I was in charge of trying to solve this problem at the company. The HLA screening idea Tony put forward almost certainly won't work to eliminate DILI risk if the peptide immunogenicity hypothesis is true. As said there are way too many HLA variants and elements that interact with HLA to mediate immunogenicity in each patient and nex-z needs to be able to conclusively rule out DILI risk in patients if its going to compete against the likely"
X Link 2025-11-05T20:28Z [----] followers, [----] engagements
"November 5th [----] Daily Update Didn't get quite as cranked today as I feared with $RARE rebounding a good bit from its after-hour lows yesterday. Think the market realized that non-dilutive financing to support setrusumab (and others in the pipeline) may actually be a good thing for shareholders. Setrusumab critical though especially as baseline revenue goes away with the OMERS deal. I think the $NKTR and $ABVX later buy positions will rebound with time. Not too worried about those $QURE is just ugly at the moment. Got worked again today after the biohaven news. I thought $PASG might hold up"
X Link 2025-11-06T02:27Z [---] followers, [---] engagements
"@david_kochman What makes you say that"
X Link 2025-11-06T20:17Z [---] followers, [--] engagements
"In just awful awful awful news the DILI patient passed away. My thoughts and prayers are with this patient may he rest in peace and his family. I believe this further raises the probability that stochastic CRISPR-cas9 indels / frameshifts can lead to either toxic mRNA or an mRNA that is able to break through nonsense mediated decay and create either a toxic or immunogenic protein. I link to a post I made where I lay out this mechanism in the second post down in this thread. This has a good chance of signaling the end of first-gen CRISPR cas9s viability. If the issue is NMD breakthrough on a"
X Link 2025-11-06T22:03Z [----] followers, [----] engagements
"Dude in good faith if youre in $NTLA I would get out. I dont think you understand the system yet. The primary goal of inducing NHEJ through cas9s DSB in a gene is to create indels that will lead to the genes mRNA being degraded through NMD. The DILI is more likely an on-target effect than an off-target and if it is given a patient died not at all safe"
X Link 2025-11-07T02:23Z [---] followers, [---] engagements
"November 6th [----] Daily Update Getting cooked for real this week. Luckily think $NKTR will see a nice rise tomorrow based on after-hours response to earnings and perhaps see more after the weekend. Single arm rare / severe disease gene therapies ( $PASG $RCKT) getting beat down post $QURE. $QURE news really rocked the thesis that the FDA would be maximally constructive in those spaces. $PEPG dipped about the lowest it has since data. $PRME doing the old plummet too probably mediated by sector effects of $NTLA. Though as said think the most likely causative feature of $NTLA's editor cas9 DSB's"
X Link 2025-11-07T02:46Z [----] followers, [----] engagements
"I really couldn't tell you. No new news. It's seemed to hold the line at $4.50 since 15mg / kg data release in the fall. It jumped up again after the $RNA buyout but figured it would come down after that as I think a buyout is just too abstract at this stage for the market. Perhaps its just low volume. They have two investor conferences next week and they have a real shot at best in class based on early data so maybe that will give it a boost. Then MAD 5mg / kg functional data is likely the next significant catalyst"
X Link 2025-11-07T02:58Z [---] followers, [---] engagements
"Its a very good question. The cas9 nuclease $NTLA uses (as does $CRSP and $EDIT) creates a double strand break in the DNA which results in the endogenous non homologous end joining repair mechanism getting activated and that repair mechanism usually ends up inserting or deleting bases at the site of the double strand break. This is a design feature of cas9 rather than a bug. The number of bases typically inserted or deleted are stochastic. Its most often 1-2 bases but can range to 50+ bases. That will typically cause a frameshift and lead to an mRNA transcript that codes for a very different"
X Link 2025-11-07T10:39Z [---] followers, [---] engagements
"I've said it before and I'll say it again $XBI"
X Link 2025-11-07T17:42Z [----] followers, [---] engagements
"This papers finding (link in comments) that cas9 induced double strand breaks drives a p53 mediated DNA damage response which yields durable markers of cellular senescence and inflammation represents another category of concern for first generation CRISPR and by extension $NTLA and $CRSP. Existing evidence makes it reasonable to think DSB effects both those described in the literature and those undescribed may have consequences that take years or decades to emerge. The extent to which these long-term effects have clinically relevant consequences for older patients with diseases like wild-type"
X Link 2025-11-10T10:55Z [----] followers, [----] engagements
"This is legitimately impressive and unprecedented efficacy data in ATTR-CM from $NTLA. If it were not for the DILI case recently reported $NTLA's stock would have traded up enormously on this news and I think all the reasonable bears would acknowledge this. If acoramidis and vutrisiran were the only game in town $NTLA might have a shot at a viable path forward on the heels of this data pending the outcome of the DILI investigation. Heart failure is a big deal and obviously kills people. So there is an ethical and rational basis for prescribing a drug that has very rare cases of DILI if it"
X Link 2025-11-10T21:55Z [----] followers, [----] engagements
"Definitely possible. I really don't have any insight into the current trading dynamics. From my perspective $PEPG still is in possession of best-in-class data for a DM1 asset at this stage the $RNA buyout was a good comp for $PEPG's upside if they can continue to demonstrate best-in-class data and I have not seen anything to warrant a change in my view at this time. However we'll see"
X Link 2025-11-10T21:57Z [---] followers, [---] engagements
"@AlexHumphries25 How are you going to definitively mitigate stochastic cas9 indels that create toxic mRNA or mRNA that breaks through nonsense mediated decay and yields an immunogenic peptide"
X Link 2025-11-10T23:01Z [---] followers, [---] engagements
"Certainly fair to point to the fact this is Phase I open-label data. However there are few unique characteristics of ATTR which I think allow one to reach stronger conclusions on efficacy on the basis of Phase [--] data than one could in other diseases. First the dissolution of the TTR tetramer into monomer and then aggregation into amyloid is about as definitively established as the proximal monocausal mechanism of TTR cardiac amyloidosis as it can get in medicine and without TTR it really shouldn't be possible to get the amyloid build-up. Two we have really definitive data from tafamidis vs."
X Link 2025-11-11T02:15Z [---] followers, [---] engagements
"Thank you for the response The preclinical studies unfortunately wouldnt be able to rule that out especially because animals have a different TTR sequence than humans so an indel induced frameshift in them doesnt generate the mRNA sequence that gets generated from an indel induced frameshift in humans and wouldnt generate the same protein. Humans also have quite a bit of TTR variation over [---] variants"
X Link 2025-11-11T09:43Z [---] followers, [--] engagements
"@BK7959925597715 Dude sorry I missed this. I know it is definitely a strange market quirk that a fire sale would almost certainly fetch well north of where it currently trades. One argument for early stage companies staying private for longerthough we wouldnt get access"
X Link 2025-11-11T11:42Z [---] followers, [--] engagements
"@BiotechObserver Its because nex-zs TTR knockdown happens much quicker and is deeper than vutrisiran or patrisiran. $ALNYs next-gen RNAi in development (nucresiran) has shown equivalent depth and speed of TTR knockdown in humans so likely will show same efficacy as nex-z"
X Link 2025-11-11T16:16Z [----] followers, [---] engagements
"@Alex___VA Yes for him I certainly agree he is disputing the efficacy. Based on the comments Ive seen I would not include him in the category of $NTLA bears who are well-informed or logical about the situation"
X Link 2025-11-11T20:19Z [---] followers, [---] engagements
"Me looking at green when Ive developed Stockholm syndrome for red"
X Link 2025-11-11T20:56Z [---] followers, [---] engagements
"November 11th [----] Daily Update Sure do love FDA rumors. 9.6% jump on the day. We'll see if it holds $XBI"
X Link 2025-11-11T21:17Z [----] followers, [---] engagements
"Little surprised I'm not seeing as much chat on $PCVX as I would have expected though perhaps it's just lost in the algorithm. It has run some from its lows but been an attractive opportunity imo"
X Link 2025-11-11T21:23Z [----] followers, [---] engagements
"November 12th [----] Daily Update BIG jump for $PASG off its recent lows. It is of course a high risk investment and may remain volatile through the 1H [----] updates. However I continue to believe the risk-reward profile here is extremely strong. To that point $LLY paid $1.04Bn (inclusive of a CVR) for Prevail and its FTD-GRN therapy back in [----] both $PASG and $LLY are in Phase [--] / [--] (though should note Prevail also had a PD asset in clinical development) and my view is that $PASG has shown more compelling data to date on both efficacy and safety. $PASG is still trading under $30M at the"
X Link 2025-11-12T21:35Z [----] followers, [----] engagements
"November 14th [----] Daily Update Little delayed in the Friday daily update post. On Friday I partially exited the $BBIO position and reallocated the proceeds towards $PRME. I will finish up a post tonight or tomorrow detailing my rationale but wanted to get this out before markets reopen for the public record"
X Link 2025-11-16T22:03Z [----] followers, [----] engagements
"I have increased my $PRME position for five reasons. First and foremost I believe it is highly likely that PM359 ( $PRME's CGD therapy) will receive FDA approval under the plausible mechanism pathway in the coming months as it definitively meets each of the five tenets of the FDA's plausible mechanism pathway. Indeed if one were to write an article explaining the rationale for approving CGD on a standalone basis I think it would look almost identical to the article Dr. Makary and Dr. Prasad published. Furthermore there are a number of pieces of circumstantial evidence that lend credence to"
X Link 2025-11-17T13:07Z [----] followers, 11.4K engagements
"November 17th [----] Daily Update. $PRME had a nice rally into the close following the increase in position size on Friday. I explained why I increased the position in a separate post. We will see if it holds. $PEPG also showed real strength today. $NKTR did as well. $ABVX continues to inch up. These four drove a gain today in the portfolio. Also opened a very small position in $IVVD at pre-market pricing. Lastly sold some more of the $BBIO position. It's a challenge for me to prioritize between positions I think are attractive amidst a finite pool of capital. I still think $BBIO can run."
X Link 2025-11-18T00:26Z [---] followers, [----] engagements
"This is a very good post by Adam. I'd like to add a couple of thoughts on AMT-130 and speak to the applicability of some of these concepts to $PASG and alpha-synuclein targeted therapies in Parkinson's Disease. First there may be an issue with tominersin in addition to the ones Adam spoke to. The huntingtin protein is absolutely essential to neuronal (and glial for that matter) function and health. This is well described in both human beings and animal models. In fact human beings born with compound heterozygous mutations in the HTT gene (one mutated copy from each parent) experience a rare"
X Link 2025-11-18T02:32Z [----] followers, 14.1K engagements
"$PASG has published data on the biodistribution of the AAV1 Vector / PGRN transgene in their animal models which should correlate to PGRN expression unless theres a question around whether the transgenes are actually driving expression. My guess is youre referring to the [----] study out of Penn. That study actually showed AAV1 transduction was widespread throughout the brain though only transduced about 1% of neurons astrocytes etc. deep to the ependymal cells in NHPs. So youre right that the meninges / ependyma are transduced in higher quantities and concentrations of PRGN in the CSF are"
X Link 2025-11-18T10:26Z [----] followers, [---] engagements
"Maybe Im misunderstanding you but what do you think is nonsense speculation here I think the vast majority of commentary here is evidenced based and any speculation is both caveated as such and reasonable. I also do think this is an important conversation. I dont see how theres any way to say that AMT-130 doesnt work and to my eye there is more evidence than any other HD therapy has shown previously that it may work. So whether current HD patients have the right to try it on the basis of existing data I think is a conversation worth having irrespective of any investment considerations. i"
X Link 2025-11-18T10:54Z [---] followers, [----] engagements
"Regarding $LLY theyre in Phase I and havent shown any human data at all yet nor substantive preclinical data. If youre concerned about $PASG not publishing ISF PGRN concentrations (just vgs) you should be particularly concerned about $LLY / Prevail in that respect. As an additional point re human data it is notable that we know of no living human with a high-confidence LoF alpha-synuclein variant which precludes the kind of MR studies that have shown benefit in most other siRNA contexts. Further studies show that in active PD outcomes are worse for patients who have lower levels of monomeric"
X Link 2025-11-18T13:00Z [----] followers, [---] engagements
"You seem to believe a PBFT02 failure is fait accompli though the basis for that appears to center on the fact that $PASG has not reported PGRN ISF concentrations that appears you presume they are hiding. It would be rather strange they would take the low-dose cohort forward if there is some unreleased internal data suggesting that is an issue. They will start to report more fulsome data in 1H [----] so we will see how that unfolds. The data from the new cohort of patients they dosed very early in disease course will be especially interesting. Whilst I fully acknowledge the high risk nature of"
X Link 2025-11-18T15:01Z [----] followers, [---] engagements
"Correct re spreading occurring. However this is where some nuance comes into play. This is actually a very different phenomenon in $QUREs case and may be a good thing as early HD pathophysiology might be broader in site of origin that just the striatum). The key thing to see with $QUREs is that profile of spread from the site of injection in the striatum is going to be a precise dose delivered to the striatum at the level you think will knock down mHTT sufficiently and then the levels of miRNA expressed will get lower as the AAV spreads out from the site of injection. So youre titrating the"
X Link 2025-11-18T18:29Z [----] followers, [---] engagements
"Whatever your views on $QURE this is actually a very important point. Whilst it is foolish to think experts don't get things wrong all the time or that a lay person who is smart and reads up on a topic can't come up with far better ideas than the so-called experts.it is similarly foolish to think the average doctor or average neurologist possesses anything approaching the breadth of HD specific domain knowledge or intellectual horsepower of Dr. Tabrizi. She is rightly considered a genuine leader in the HD field Doesn't mean she is guaranteed to be right here and differing viewpoints should be"
X Link 2025-11-19T00:09Z [----] followers, 19.1K engagements
"November 18th [----] Daily Update $PEPG continues to rip on though backed off some at the end of the day. $PRME comes back down from the end of the day jump yesterday"
X Link 2025-11-19T00:13Z [---] followers, [----] engagements
"I think an HD patient has the right to make that call for themselves under informed consent. I am not so sure you would have the same views on this if you were staring down the certainty of a horrible loss of your memory reasoning capabilities motor function and ability to take care of yourself leading to an early death. If I were an HD patient and at an age where I was likely to convert from asymptomatic to symptomatic I would personally choose taking this shot. I will note one of those three patients had an CSF leak due to an LP required as part of the trial to do assays on the CSF."
X Link 2025-11-19T11:17Z [---] followers, [---] engagements
"Added remaining cash from exits to $JANX. Janx now about 5% of portfolio"
X Link 2025-12-01T15:40Z [----] followers, [----] engagements
"Further exited the $RARE position to add to $JANX. Full breakdown posted tonight. However want to publicly disclose for accountability given post-market update"
X Link 2025-12-01T16:15Z [----] followers, [----] engagements
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