[GUEST ACCESS MODE: Data is scrambled or limited to provide examples. Make requests using your API key to unlock full data. Check https://lunarcrush.ai/auth for authentication information.] #  @RNAiAnalyst Dirk Haussecker Sarepta Therapeutics ($SRPT) has received positive news regarding its gene therapy, Elevidys, as the FDA has lifted a hold on the treatment. The company's subcutaneous delivery of a Huntington's disease candidate has shown impressive results, although it does not target exon X. Additionally, Sarepta's partnership with Arrowhead Pharmaceuticals ($ARWR) may be renegotiated following the recent news. ### Engagements: XXXXXX [#](/creator/twitter::409194010/interactions)  - X Week XXXXXXX -XX% - X Month XXXXXXX +61% - X Months XXXXXXXXX +64% - X Year XXXXXXXXX +2% ### Mentions: XX [#](/creator/twitter::409194010/posts_active)  - X Week XX -XXXX% - X Month XXX +41% - X Months XXX +30% - X Year XXXXX +27% ### Followers: XXXXXX [#](/creator/twitter::409194010/followers)  - X Week XXXXXX +0.39% - X Month XXXXXX +2.30% - X Months XXXXXX +8.40% - X Year XXXXXX +11% ### CreatorRank: XXXXXXX [#](/creator/twitter::409194010/influencer_rank)  ### Social Influence [#](/creator/twitter::409194010/influence) --- **Social category influence** [stocks](/list/stocks) XXXXX% [finance](/list/finance) XXXX% [cryptocurrencies](/list/cryptocurrencies) XXXX% [countries](/list/countries) XXXX% **Social topic influence** [$srpt](/topic/$srpt) #31, [$qure](/topic/$qure) #15, [$rna](/topic/$rna) #1, [$pepg](/topic/$pepg) #1, [$ions](/topic/$ions) #1, [$arwr](/topic/$arwr) #4, [$alny](/topic/$alny) #1, [$mgx](/topic/$mgx) #1, [$ntla](/topic/$ntla) #12, [$dyn](/topic/$dyn) #1 **Top accounts mentioned or mentioned by** [@geneinvesting](/creator/undefined) [@grok](/creator/undefined) [@bambossie81](/creator/undefined) [@djtblows69](/creator/undefined) [@hall8jack](/creator/undefined) [@drmakaryfda](/creator/undefined) [@adamfeuerstein](/creator/undefined) [@towiu2](/creator/undefined) [@jfais20](/creator/undefined) [@hulksmash8181](/creator/undefined) [@john_hersc79276](/creator/undefined) [@biopharmiq](/creator/undefined) [@zhaoweiasu](/creator/undefined) [@jamesmclaw70633](/creator/undefined) [@pearlf](/creator/undefined) [@elilillyandco](/creator/undefined) [@sonichedgefund](/creator/undefined) [@hannibalspeaks](/creator/undefined) [@alamentarius](/creator/undefined) [@usfda](/creator/undefined) **Top assets mentioned** [Sarepta Therapeutics, Inc. (SRPT)](/topic/$srpt) [uniQure N.V. (QURE)](/topic/$qure) [Avidity Biosciences, Inc. Common Stock (RNA)](/topic/$rna) [Ionis Pharmaceuticals, Inc. Common Stock (IONS)](/topic/$ions) [Arrowhead Research Corporation (ARWR)](/topic/$arwr) [Alnylam Pharmaceuticals, Inc. (ALNY)](/topic/$alny) [Intellia Therapeutics, Inc (NTLA)](/topic/$ntla) [Dyne Therapeutics, Inc. Common Stock (DYN)](/topic/$dyn) [Scholar Rock Holding Corporation Common Stock (SRRK)](/topic/$srrk) [Regeneron Pharmaceuticals Inc (REGN)](/topic/$regn) [Vertex Protocol (VRTX)](/topic/$vrtx) [Biogen Inc (BIIB)](/topic/$biib) [Synthetify (SNY)](/topic/$sny) [Smart Layer Network (SLN)](/topic/$sln) [BridgeBio Pharma, Inc. Common Stock (BBIO)](/topic/$bbio) [Novavax Inc (NVAX)](/topic/$nvax) [Moderna Inc (MRNA)](/topic/$mrna) [Mettalex (MTLX)](/topic/$mtlx) [MoonLake Immunotherapeutics Class A Ordinary Shares (MLTX)](/topic/$mltx) [Novo-Nordisk (NVO)](/topic/$nvo) [Akropolis (AKRO)](/topic/$akro) ### Top Social Posts [#](/creator/twitter::409194010/posts) --- Top posts by engagements in the last XX hours "$ntla $srpt cant see Adam's tweets but is he squealing yet Do his handlers need to up the retainer" [X Link](https://x.com/RNAiAnalyst/status/1975969912473649393) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-08T17:01Z 20.5K followers, 8454 engagements "$MGX tomorrow is $IONS R&D day. Maybe they will grace them with a mention. Must be the only pure-play #CRISPR stock trading at XX% discount. Today Algen-AstraZeneca CRISPR functional genomics deal involving AI. Metagenomi maybe hottest CRISPR-AI stock out there" [X Link](https://x.com/RNAiAnalyst/status/1975220838501920810) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-06T15:25Z 20.5K followers, 3055 engagements "$rna for the boring biotech traders on this app this one could be for you. I don't see how this does not get acquired over next year. Their delivery tech is legit and trailblazing. Initially thought conjugating oligos to antibodies too clumsy.but they made it work. Just don't get chickened out when buyout rumor comes and goes" [X Link](https://x.com/RNAiAnalyst/status/1974197621230055839) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-03T19:39Z 20.5K followers, 5682 engagements "$qure $pepg today we are seeing groundbreaking data for X of the most common severe monogenic diseases: Huntington's and myotonic dystrophy type X. A XXXX% splicing correction hints at a cure for muscle-related issues in DM1; for Huntington's exon 1-knockdown will ultimately be complemented by strategies to slow down triplett expansion" [X Link](https://x.com/RNAiAnalyst/status/1970963092935770452) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-09-24T21:26Z 20.5K followers, 11.2K engagements "$dyn revisiting shift of primary endpoint from CASI (splicing score) to vHOT (functional endpoint) as demanded by new #FDA: what happened to 'plausible biological pathway' Myotonia affects many but not all DM1 patients + various degrees mis-splicing is common to all forms of DM1. $rna $pepg" [X Link](https://x.com/RNAiAnalyst/status/1956018913981907022) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-08-14T15:43Z 20.5K followers, 2495 engagements "50% huntingtin exon X kd better than XX% full-length. $qure" [X Link](https://x.com/RNAiAnalyst/status/1977401217828401476) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-12T15:49Z 20.5K followers, 10.2K engagements "$srpt why would this boy receive systemic AAV gene therapy at time when he had elevated liver labs Why" [X Link](https://x.com/RNAiAnalyst/status/1973790163408187844) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-02T16:40Z 20.5K followers, 12.1K engagements "$ions first clinical RNAi data to be presented (semiannual ApoC3) and a Dravet antisense oligo). $arwr $stok Nothing on $mgx partnership in press release" [X Link](https://x.com/RNAiAnalyst/status/1975518860951392279) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-07T11:09Z 20.5K followers, 7232 engagements "$NTLA I believe the ATTR-PN phase X data will be even better than the one reported today mainly because p3 does not include patients that had progressed on $ALNY's RNAi agents before. As you can see they did worse than knockdown-naive subjects" [X Link](https://x.com/RNAiAnalyst/status/1971291795968622899) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-09-25T19:12Z 20.5K followers, 4225 engagements "$ALNY presents potentially game-changing argument for why ATTR-CM patients should take a silencer and not a stabilizer. -- vutrisiran reduces gastrointestinal events REGARDLESS of 1) tafamidis (which does not reduce baseline gastro effects) and 2) mutant or wild-type ATTR-CM. This is a reminder that ATTR is a multi-system disease and stabilizers possibly only good for cardiomyopathy manifestations. Important readthrough to $BBIO and $NTLA" [X Link](https://x.com/RNAiAnalyst/status/1972721472612216934) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-09-29T17:53Z 20.5K followers, 10.5K engagements "$qure amt-130 presentation about to conclude. Anything new biotwitter" [X Link](https://x.com/RNAiAnalyst/status/1977401994030510275) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-12T15:52Z 20.5K followers, 15.5K engagements "Most important slide. Note how soon bilirubin moves.quite different from non-fatal ALIs. Question for AAV aficionados: is there similar pattern in past cases of systemic AAV ALFs $srpt" [X Link](https://x.com/RNAiAnalyst/status/1977023845153624178) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-11T14:49Z 20.5K followers, 10.8K engagements "More success for locally delivered #AAV gene therapy. $qure $regn $alsen.pa $bntc" [X Link](https://x.com/RNAiAnalyst/status/1977439464655085702) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-12T18:21Z 20.5K followers, 9818 engagements "$pepg sh*t if you thought $qure was crazy good look at Pepgen's 15mg/kg data XXXX% splicing correction basically twice the competition including $RNA. Not DMPK knockdown but disruption of pathogenic RNA structure. Differentiation Only $VRTX similar but earlier-stage approach.and I like $pepg's approach better than $VRTX. Crazy day got even better. $dyn $90M market cap" [X Link](https://x.com/RNAiAnalyst/status/1970957537160855735) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-09-24T21:04Z 20.5K followers, 35.6K engagements "@GeneInvesting @zhaoweiasu @ArkadiBaudelair The larger issue with wtATTR-CM is proper diagnosis. You obviously dont want to spend $2M on a patient that does not have TTR-related issues. $alny and $bbio risking a backlash if current trends continue" [X Link](https://x.com/RNAiAnalyst/status/1969017654322065505) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-09-19T12:36Z 20.5K followers, XXX engagements "$dyne sometimes when you are at the conference u are last to see data release PR. Saw the DM1 data this morning and was impressed by continued improvements and CNS-related symptom benefits. $rna $pepg" [X Link](https://x.com/RNAiAnalyst/status/1975850170433818797) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-08T09:06Z 20.5K followers, 2760 engagements "$PEPG disrupts myotonic dystrophy type X (blog): $RNA $DYNE $VRTX $TRDA" [X Link](https://x.com/RNAiAnalyst/status/1971128732049080715) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-09-25T08:24Z 20.5K followers, 4049 engagements "Plea to $srpt and $arwr: Just Focus on Aberrant Exon X Transcript in Huntingtons Disease (blog): $qure" [X Link](https://x.com/RNAiAnalyst/status/1972940608840081913) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-09-30T08:24Z 20.5K followers, 6743 engagements "$SRRK 'if I do wanna be on treatment I NOW HAVE THAT OPTION'. I doubt she is referring to the $BIIB drug" [X Link](https://x.com/RNAiAnalyst/status/1970223028559847693) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-09-22T20:25Z 20.5K followers, 5209 engagements "$EDIT just read up on LDL-R upregulation data in NHP.90-98% LDL-c reduction WITH EASE. A program to watch" [X Link](https://x.com/RNAiAnalyst/status/1978442686387368189) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-15T12:47Z 20.5K followers, 20.4K engagements "$srpt convened an expert panel on enhancing safety of Elevdiys. Observations: in both cases of ALF liver enzymes first increased X weeks post dose death 11weeks post dose (predictable time-course). #bilirubin was not increased in both fatalities shortly after liver enzyme increases vs no such observations in the XX non-fatal ALIs No relationship with weight/dose but likely liver health (steatosis) and consumptive coagulopathy may ultimately cause of T cell-initiated fatal course. Recs: more sensitive monitoring of liver tox markers; TREAT at earliest sign of liver enzyme increase with" [X Link](https://x.com/RNAiAnalyst/status/1976972698564575558) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-11T11:26Z 20.5K followers, 16.8K engagements "$60B MC $alny's biz dev team needs to be summarily fired for not taking advantage of #CRISPR firesale prices not long ago. CRISPR $ntla will eat a lot of its market share in the not too distant future. What were they thinking" [X Link](https://x.com/RNAiAnalyst/status/1975988277321793649) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-08T18:14Z 20.5K followers, 4820 engagements "In addition to knocking down huntingtin exon X we need additional strategies aiming at lowering use of cryptic intron X polyA sites (e.g. promoting RNAPolII processivity; targeting polyA signals using oligos etc). $qure $arwr $ions $srpt $wve #Huntingtons" [X Link](https://x.com/RNAiAnalyst/status/1977333164075802645) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-12T11:18Z 20.5K followers, 2882 engagements "Prediction: $ALNY will acquire RNAi player $RNA. DM1 is a similar-sized opportunity with ATTR-CM. $alny can make case of developing next-gen DM1 and FSHD medicines plus new muscle franchise" [X Link](https://x.com/RNAiAnalyst/status/1978841255783063872) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-16T15:11Z 20.5K followers, 4939 engagements "@john_hersc79276 In a disease where progression is so variable from patient to patient unless you run a much larger trial propensity matching much more suitable than random placebo. Of course you could ask for such a trial but have patients wait another X years. $qure" [X Link](https://x.com/RNAiAnalyst/status/1976368769560871365) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-09T19:26Z 20.5K followers, XXX engagements "$mgx if they continue to make hemophilia A their priority (I'd advise against) then aiming for BTD based on preclinical data should be their goal :). $sprb" [X Link](https://x.com/RNAiAnalyst/status/1975312710515777651) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-06T21:30Z 20.5K followers, 3486 engagements "$srpt looks like next Saturday there could be a pretty important poster on sirolimus use with elevidys at WMS25:" [X Link](https://x.com/RNAiAnalyst/status/1973500468065345843) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-01T21:29Z 20.5K followers, 15.3K engagements "$qure Sunday for first scientific AMT-130 pivotal trial presentation" [X Link](https://x.com/RNAiAnalyst/status/1976732939020124200) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-10T19:33Z 20.5K followers, 24.9K engagements "$ntla this Biocryst acquisition of $atxs made my day. First claimimf that patients CLEARLY prefer oral for prophylaxis now saying 'sorry we lied to you: every X months and less often much better'. Clearly freaking out about the coming one-time cure. $bcrx" [X Link](https://x.com/RNAiAnalyst/status/1978103253142237254) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-14T14:19Z 20.5K followers, 6952 engagements "@GeneInvesting 100k in US/EU each. Highly underdiagnosed. Could be more. Partly trolling $ALNY but for their own good. I'm just an internet guy ;)" [X Link](https://x.com/RNAiAnalyst/status/1978843218553848051) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-16T15:19Z 20.5K followers, XXX engagements "$mgx remember this pure-play CRISPR Co (partnered with $IONS) trades XX% BELOW CASH" [X Link](https://x.com/RNAiAnalyst/status/1978846977975525447) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-16T15:34Z 20.5K followers, 3423 engagements "@richtrades100 Of course it is about color $srpt provide about current market dynamics plus progress on successfully implementing sirolimus" [X Link](https://x.com/RNAiAnalyst/status/1976218711997796448) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-09T09:30Z 20.5K followers, XXX engagements "Anybody knows whether 'Dear Leader' got an mRNA or the $nvax protein covid vax $bntx $mrna" [X Link](https://x.com/RNAiAnalyst/status/1976967600807834104) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-11T11:06Z 20.5K followers, 5594 engagements "$srpt adds $200M to cash balance in Q2 alone. Balance now at $850M" [X Link](https://x.com/RNAiAnalyst/status/1953189807431270865) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-08-06T20:21Z 20.5K followers, 38.9K engagements "$ntla believe the next major re-rating of stock occurs when commercial uptake shows patients/KOLs love that type of medicine" [X Link](https://x.com/RNAiAnalyst/status/1978197853127180461) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-14T20:34Z 20.5K followers, 5485 engagements "$NTLA unlike vutrisiran mNIS+7 improves in majority of ATTR-PN patients after NTLA-2001 one time treatment. Another @NEJM publication in the bag. $alny" [X Link](https://x.com/RNAiAnalyst/status/1971219595408310791) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-09-25T14:25Z 20.5K followers, 3792 engagements "$pepg stock is held almost exclusively by tutes e.g. RA Capital (32%) Point72 (8%); I seem to be the only tax-paying non-tute 😜. Regardless considering $RNA and $DYN market caps of $6B and $2B respectively would be unfair to see offering at anything less than $300M pre-money. $pepg should have announced data first then do an offering after full trading day. Maybe RA Capital wants to add more and that's why" [X Link](https://x.com/RNAiAnalyst/status/1970961941343715457) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-09-24T21:22Z 20.5K followers, 6352 engagements "$WVE AATD was never the big money maker for Co. It validated the #RNAediting platform. For most applications XX% editing is plenty. Yet it sold off on $BEAM/repeat admin data when obesity and cardiovascular are the crown jewels. Forget about Huntington's and DMD" [X Link](https://x.com/RNAiAnalyst/status/1976390489084493907) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-09T20:53Z 20.5K followers, 3535 engagements "$qure IMO the coming intrathecal RNAi competition need to show superiority to one-time AMT-130. Life-long progressive disease. $alny $arwr" [X Link](https://x.com/RNAiAnalyst/status/1971485227894833480) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-09-26T08:01Z 20.5K followers, 4052 engagements "💪week: $RNA (also M&A rumors) $DYN (pin action+WMS) $PEPG $SRPT (WMS25). $qure AMT-130 data presentation" [X Link](https://x.com/RNAiAnalyst/status/1974756848688591306) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-05T08:41Z 20.5K followers, 4502 engagements "$SNY be it atopic dermatitis hidradenitis suppuritiva etc.p2 -- p3 results measuring improvement from baseline can differ greatly depending on patient pop (severe/moderate) geography (SOC) etc $NKTR $MTLX" [X Link](https://x.com/RNAiAnalyst/status/1963584805229990333) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-09-04T12:47Z 20.5K followers, 2292 engagements "$ALNY $REGN: The POWER of #genetic medicine illustrated: (complement C5) mono RNAi better than RNAi-mAb combo despite XX% vs XX% complement assay inhibition respectively for myasthenia gravis. -- why I also expect $NTLA's ATTR and HAE treatment to kick ass. $SLN" [X Link](https://x.com/RNAiAnalyst/status/1960333599183696078) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-08-26T13:28Z 20.5K followers, 6110 engagements "A backup site What a revolutionary idea. $regn $srrk" [X Link](https://x.com/RNAiAnalyst/status/1975858209182154926) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-08T09:38Z 20.5K followers, 4011 engagements "$abvx in presentation CEO commended team on keeping placebo response low (by limiting enrolment in certain geographies etc) pointing to other recent examples i.e. $mltx and $sny. We all know these'failed' drugs are all active; most of the game is about pbo response mgmt" [X Link](https://x.com/RNAiAnalyst/status/1978472361184927943) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-15T14:45Z 20.5K followers, 6554 engagements "Seems like all the #biotech M&A's get preannounced by the FT or WSJ on Sundays now. Suddenly regret that I was not more aggressive on $RNA in after-hours. My bids did not get filled at $47.8" [X Link](https://x.com/RNAiAnalyst/status/1974461816672817172) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-04T13:09Z 20.5K followers, 5598 engagements "@minimus0987 @SuperDuperDrugs @sonichedgefund @grok CEO said X weeks ago that FDA already saw and reviewed the 240mg dose expansion data incl safety and knockdown and greenlighted 400mg. $wve" [X Link](https://x.com/RNAiAnalyst/status/1976654941445116276) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-10T14:23Z 20.5K followers, XXX engagements "$srpt 2027 convertible debt burden down from $1.2B to $450M with remainder largerly deferred to 2030: Seems like bankruptcy a highly unlikely scenario now and full steam ahead with #RNAi pipeline. $arwr" [X Link](https://x.com/RNAiAnalyst/status/1958488907894661520) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-08-21T11:18Z 20.5K followers, 11.3K engagements "$srrk $regn smart resourceful traders probably have a list with upcoming PDUFAs for new formulations that rely on the Catalent site. Can I see this list Catalent insiders probably shorting all of them now" [X Link](https://x.com/RNAiAnalyst/status/1977780133915189686) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-13T16:55Z 20.5K followers, 6552 engagements "$10B.that is the combined market cap of major DM1 players $RNA $DYN and $PEPG; add to this $VRTX w/ a approach and stage similar to $PEPG. $RNA also prices in large FSHD opportunity. DM1 is one of the largest genetic disease categories larger than CF. Combined market cap could ultimately be $50B in 2029. There is also likely a succession of therapies coming onto market that improve on earlier approved drugs: 1) $RNA: most advanced evidence of fct improvements but probably least effective on molecular level (CASI) 2) $DYN: a year behind $RNA but CASI superior and impressive fct data at WMS25" [X Link](https://x.com/RNAiAnalyst/status/1978743257090642124) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-16T08:42Z 20.5K followers, 4509 engagements "$prqr I will jinx it but the only explanation for not hearing anything from the company would be M&A. Anything else would be reason for panic. Will have to see whether $nvo jas some leftovers in poxkets after today's $akro acquisition" [X Link](https://x.com/RNAiAnalyst/status/1976374585684787578) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-09T19:49Z 20.5K followers, 3646 engagements "$BIIB $IONS CRL issued for another SMA drug today high-dose nusinersen; and once again it is because of CMC" [X Link](https://x.com/RNAiAnalyst/status/1970590861407985885) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-09-23T20:47Z 20.5K followers, 5573 engagements "$RNA moving after-hours. Acquisition to be announced ahead of WMS25 Speculating is OK no" [X Link](https://x.com/RNAiAnalyst/status/1974205410543341696) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-03T20:10Z 20.5K followers, 4930 engagements "Down-to-earth data-based discussion of $pepg's DM1 program safety and efficacy. $dyn $rna" [X Link](https://x.com/RNAiAnalyst/status/1978588482315378977) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-15T22:27Z 20.5K followers, 8240 engagements "@Zappitelli1 No way. I'd be terrified having a significant long position going into earnings. Sandy has strung along shareholders quarter after quarter.and he'll continue to do so likely with another highly dilutive financing. $sgmo" [X Link](https://x.com/RNAiAnalyst/status/1952791488343216314) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-08-05T17:59Z 20.5K followers, 1204 engagements "@anna51296106 $srpt is now studying sirolimus in non-ambulatory. My guess is that if safety positive then back on label h2 2026. $srpt" [X Link](https://x.com/RNAiAnalyst/status/1976986829141512425) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-11T12:22Z 20.5K followers, 1580 engagements "$SLN may be the first RNAi casualty due to competition from China. This Co has done X over last decade in terms of progressing their tech incl extrahepatic delivery as more+more partnering competition for Lpa complement etc emerges" [X Link](https://x.com/RNAiAnalyst/status/1961437276828839985) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-08-29T14:34Z 20.5K followers, 2378 engagements "$qure Seems that AMT-130 availability could be less than demand initially but I expect that in profit-driven healthcare system hospitals will recognize potential of $clpt-enabled procedures and invest accordingly. 'The most important part of the surgery length beyond people occupying an extremely rare intraoperative MRI scanner are the people who have to be in that room.'" [X Link](https://x.com/RNAiAnalyst/status/1974780770733490676) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-05T10:16Z 20.5K followers, 9374 engagements "$srpt no sirolimus PROPHYLAXIS X of XX with acute liver injury. Sirolimus prophylaxis: zero of X. Proph well tolerated. Single center experience" [X Link](https://x.com/RNAiAnalyst/status/1975894253953004000) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-08T12:01Z 20.5K followers, 12.3K engagements "This guy nailed it. FILL-FINISH.I remember @JMaraganore X decades ago stressing that work not done until fill-finish secured. The irony is that Askshay Vaishnaw went from $ALNY to $SRRK" [X Link](https://x.com/RNAiAnalyst/status/1977732814633087069) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-13T13:47Z 20.5K followers, 6418 engagements "While I see $pepg leading in terms of likely muscle benefits in DM1 remains to be seen how non-muscle symptoms with this cationic peptide conjugate improve relative to the tfr1-targeted antibody conjugates of $rna and $dyne. Similar with $arwr $srpt DM1" [X Link](https://x.com/RNAiAnalyst/status/1975850822560055703) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-08T09:08Z 20.5K followers, 3280 engagements "Decision was last week @statnews reports this morning after new bullish Elevidys data out. Mmh. $srpt" [X Link](https://x.com/RNAiAnalyst/status/1975882188139950193) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-08T11:13Z 20.5K followers, 12.7K engagements "Incredible (and timely) summary on critical importance of EXON1 in #Huntington's disease by unsung heroine Gillian Bates: -- in mouse models XX% exon 1-only kd much more impactful on pathopysiology than XX% full-length kd and much more: $qure $ions $wve $srpt $arwr" [X Link](https://x.com/RNAiAnalyst/status/1977332395641581710) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-12T11:15Z 20.5K followers, 9845 engagements "$mgx moving" [X Link](https://x.com/RNAiAnalyst/status/1978846540056711359) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-16T15:32Z 20.5K followers, 2500 engagements "There is a slide that 'based on current assumptions' there would be a genome editing clinical candidate this year $ions $mgx" [X Link](https://x.com/RNAiAnalyst/status/1975548956026544364) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-07T13:09Z 20.5K followers, 1829 engagements "$srpt getting more and more bullish ahead of earnings: 1) no more deaths since ambulatory only 2) sirolimus as backup (will have to see whether prophylaxis or on-demand wins out) 3) current market would support a $srpt pipeline even without Elevidys" [X Link](https://x.com/RNAiAnalyst/status/1975989905487757494) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-08T18:21Z 20.5K followers, 11.7K engagements "$qure after the initial euphoria and acknowledging the credibility of KOL Tabrizi and Wild can sense a heated bull-bear battle bubbling up. Think $srpt Elevidys. There are vested interests that dont like AMT-130. I also predict that Adam F will become their spokesperson" [X Link](https://x.com/RNAiAnalyst/status/1977141339281313898) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-11T22:36Z 20.5K followers, 6614 engagements "Small #CRISPR platform Co (see also $MGX) Mammoth Biosciences showcases ApoC3 disruption data in NHPs. XX% ApoC3 lowering. Wants to enter clinic for FCS in 2026 to compete with $IONS $ARWR. Looks like nuclease-based. I would recommend base or prime also for gene inhibition" [X Link](https://x.com/RNAiAnalyst/status/1976389811872125192) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-09T20:50Z 20.5K followers, 3339 engagements "@Anonym64382296 @JMaraganore Not on the yahoo feed nor an SEC filing. Had to find this on their website: $SRRK" [X Link](https://x.com/RNAiAnalyst/status/1977736713184088311) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-13T14:02Z 20.5K followers, XXX engagements "#SMA there is no apparent disadvantage from treating CNS only after intrathecal ASO (nusinersen) admin vs systemic small molecule (risdiplam). Very comprehensive analysis. $ions $biib $ptct" [X Link](https://x.com/RNAiAnalyst/status/1975870890593399051) [@RNAiAnalyst](/creator/x/RNAiAnalyst) 2025-10-08T10:28Z 20.5K followers, 2643 engagements
[GUEST ACCESS MODE: Data is scrambled or limited to provide examples. Make requests using your API key to unlock full data. Check https://lunarcrush.ai/auth for authentication information.]
Sarepta Therapeutics ($SRPT) has received positive news regarding its gene therapy, Elevidys, as the FDA has lifted a hold on the treatment. The company's subcutaneous delivery of a Huntington's disease candidate has shown impressive results, although it does not target exon X. Additionally, Sarepta's partnership with Arrowhead Pharmaceuticals ($ARWR) may be renegotiated following the recent news.
Social category influence stocks XXXXX% finance XXXX% cryptocurrencies XXXX% countries XXXX%
Social topic influence $srpt #31, $qure #15, $rna #1, $pepg #1, $ions #1, $arwr #4, $alny #1, $mgx #1, $ntla #12, $dyn #1
Top accounts mentioned or mentioned by @geneinvesting @grok @bambossie81 @djtblows69 @hall8jack @drmakaryfda @adamfeuerstein @towiu2 @jfais20 @hulksmash8181 @john_hersc79276 @biopharmiq @zhaoweiasu @jamesmclaw70633 @pearlf @elilillyandco @sonichedgefund @hannibalspeaks @alamentarius @usfda
Top assets mentioned Sarepta Therapeutics, Inc. (SRPT) uniQure N.V. (QURE) Avidity Biosciences, Inc. Common Stock (RNA) Ionis Pharmaceuticals, Inc. Common Stock (IONS) Arrowhead Research Corporation (ARWR) Alnylam Pharmaceuticals, Inc. (ALNY) Intellia Therapeutics, Inc (NTLA) Dyne Therapeutics, Inc. Common Stock (DYN) Scholar Rock Holding Corporation Common Stock (SRRK) Regeneron Pharmaceuticals Inc (REGN) Vertex Protocol (VRTX) Biogen Inc (BIIB) Synthetify (SNY) Smart Layer Network (SLN) BridgeBio Pharma, Inc. Common Stock (BBIO) Novavax Inc (NVAX) Moderna Inc (MRNA) Mettalex (MTLX) MoonLake Immunotherapeutics Class A Ordinary Shares (MLTX) Novo-Nordisk (NVO) Akropolis (AKRO)
Top posts by engagements in the last XX hours
"$ntla $srpt cant see Adam's tweets but is he squealing yet Do his handlers need to up the retainer"
X Link @RNAiAnalyst 2025-10-08T17:01Z 20.5K followers, 8454 engagements
"$MGX tomorrow is $IONS R&D day. Maybe they will grace them with a mention. Must be the only pure-play #CRISPR stock trading at XX% discount. Today Algen-AstraZeneca CRISPR functional genomics deal involving AI. Metagenomi maybe hottest CRISPR-AI stock out there"
X Link @RNAiAnalyst 2025-10-06T15:25Z 20.5K followers, 3055 engagements
"$rna for the boring biotech traders on this app this one could be for you. I don't see how this does not get acquired over next year. Their delivery tech is legit and trailblazing. Initially thought conjugating oligos to antibodies too clumsy.but they made it work. Just don't get chickened out when buyout rumor comes and goes"
X Link @RNAiAnalyst 2025-10-03T19:39Z 20.5K followers, 5682 engagements
"$qure $pepg today we are seeing groundbreaking data for X of the most common severe monogenic diseases: Huntington's and myotonic dystrophy type X. A XXXX% splicing correction hints at a cure for muscle-related issues in DM1; for Huntington's exon 1-knockdown will ultimately be complemented by strategies to slow down triplett expansion"
X Link @RNAiAnalyst 2025-09-24T21:26Z 20.5K followers, 11.2K engagements
"$dyn revisiting shift of primary endpoint from CASI (splicing score) to vHOT (functional endpoint) as demanded by new #FDA: what happened to 'plausible biological pathway' Myotonia affects many but not all DM1 patients + various degrees mis-splicing is common to all forms of DM1. $rna $pepg"
X Link @RNAiAnalyst 2025-08-14T15:43Z 20.5K followers, 2495 engagements
"50% huntingtin exon X kd better than XX% full-length. $qure"
X Link @RNAiAnalyst 2025-10-12T15:49Z 20.5K followers, 10.2K engagements
"$srpt why would this boy receive systemic AAV gene therapy at time when he had elevated liver labs Why"
X Link @RNAiAnalyst 2025-10-02T16:40Z 20.5K followers, 12.1K engagements
"$ions first clinical RNAi data to be presented (semiannual ApoC3) and a Dravet antisense oligo). $arwr $stok Nothing on $mgx partnership in press release"
X Link @RNAiAnalyst 2025-10-07T11:09Z 20.5K followers, 7232 engagements
"$NTLA I believe the ATTR-PN phase X data will be even better than the one reported today mainly because p3 does not include patients that had progressed on $ALNY's RNAi agents before. As you can see they did worse than knockdown-naive subjects"
X Link @RNAiAnalyst 2025-09-25T19:12Z 20.5K followers, 4225 engagements
"$ALNY presents potentially game-changing argument for why ATTR-CM patients should take a silencer and not a stabilizer. -- vutrisiran reduces gastrointestinal events REGARDLESS of 1) tafamidis (which does not reduce baseline gastro effects) and 2) mutant or wild-type ATTR-CM. This is a reminder that ATTR is a multi-system disease and stabilizers possibly only good for cardiomyopathy manifestations. Important readthrough to $BBIO and $NTLA"
X Link @RNAiAnalyst 2025-09-29T17:53Z 20.5K followers, 10.5K engagements
"$qure amt-130 presentation about to conclude. Anything new biotwitter"
X Link @RNAiAnalyst 2025-10-12T15:52Z 20.5K followers, 15.5K engagements
"Most important slide. Note how soon bilirubin moves.quite different from non-fatal ALIs. Question for AAV aficionados: is there similar pattern in past cases of systemic AAV ALFs $srpt"
X Link @RNAiAnalyst 2025-10-11T14:49Z 20.5K followers, 10.8K engagements
"More success for locally delivered #AAV gene therapy. $qure $regn $alsen.pa $bntc"
X Link @RNAiAnalyst 2025-10-12T18:21Z 20.5K followers, 9818 engagements
"$pepg sh*t if you thought $qure was crazy good look at Pepgen's 15mg/kg data XXXX% splicing correction basically twice the competition including $RNA. Not DMPK knockdown but disruption of pathogenic RNA structure. Differentiation Only $VRTX similar but earlier-stage approach.and I like $pepg's approach better than $VRTX. Crazy day got even better. $dyn $90M market cap"
X Link @RNAiAnalyst 2025-09-24T21:04Z 20.5K followers, 35.6K engagements
"@GeneInvesting @zhaoweiasu @ArkadiBaudelair The larger issue with wtATTR-CM is proper diagnosis. You obviously dont want to spend $2M on a patient that does not have TTR-related issues. $alny and $bbio risking a backlash if current trends continue"
X Link @RNAiAnalyst 2025-09-19T12:36Z 20.5K followers, XXX engagements
"$dyne sometimes when you are at the conference u are last to see data release PR. Saw the DM1 data this morning and was impressed by continued improvements and CNS-related symptom benefits. $rna $pepg"
X Link @RNAiAnalyst 2025-10-08T09:06Z 20.5K followers, 2760 engagements
"$PEPG disrupts myotonic dystrophy type X (blog): $RNA $DYNE $VRTX $TRDA"
X Link @RNAiAnalyst 2025-09-25T08:24Z 20.5K followers, 4049 engagements
"Plea to $srpt and $arwr: Just Focus on Aberrant Exon X Transcript in Huntingtons Disease (blog): $qure"
X Link @RNAiAnalyst 2025-09-30T08:24Z 20.5K followers, 6743 engagements
"$SRRK 'if I do wanna be on treatment I NOW HAVE THAT OPTION'. I doubt she is referring to the $BIIB drug"
X Link @RNAiAnalyst 2025-09-22T20:25Z 20.5K followers, 5209 engagements
"$EDIT just read up on LDL-R upregulation data in NHP.90-98% LDL-c reduction WITH EASE. A program to watch"
X Link @RNAiAnalyst 2025-10-15T12:47Z 20.5K followers, 20.4K engagements
"$srpt convened an expert panel on enhancing safety of Elevdiys. Observations: in both cases of ALF liver enzymes first increased X weeks post dose death 11weeks post dose (predictable time-course). #bilirubin was not increased in both fatalities shortly after liver enzyme increases vs no such observations in the XX non-fatal ALIs No relationship with weight/dose but likely liver health (steatosis) and consumptive coagulopathy may ultimately cause of T cell-initiated fatal course. Recs: more sensitive monitoring of liver tox markers; TREAT at earliest sign of liver enzyme increase with"
X Link @RNAiAnalyst 2025-10-11T11:26Z 20.5K followers, 16.8K engagements
"$60B MC $alny's biz dev team needs to be summarily fired for not taking advantage of #CRISPR firesale prices not long ago. CRISPR $ntla will eat a lot of its market share in the not too distant future. What were they thinking"
X Link @RNAiAnalyst 2025-10-08T18:14Z 20.5K followers, 4820 engagements
"In addition to knocking down huntingtin exon X we need additional strategies aiming at lowering use of cryptic intron X polyA sites (e.g. promoting RNAPolII processivity; targeting polyA signals using oligos etc). $qure $arwr $ions $srpt $wve #Huntingtons"
X Link @RNAiAnalyst 2025-10-12T11:18Z 20.5K followers, 2882 engagements
"Prediction: $ALNY will acquire RNAi player $RNA. DM1 is a similar-sized opportunity with ATTR-CM. $alny can make case of developing next-gen DM1 and FSHD medicines plus new muscle franchise"
X Link @RNAiAnalyst 2025-10-16T15:11Z 20.5K followers, 4939 engagements
"@john_hersc79276 In a disease where progression is so variable from patient to patient unless you run a much larger trial propensity matching much more suitable than random placebo. Of course you could ask for such a trial but have patients wait another X years. $qure"
X Link @RNAiAnalyst 2025-10-09T19:26Z 20.5K followers, XXX engagements
"$mgx if they continue to make hemophilia A their priority (I'd advise against) then aiming for BTD based on preclinical data should be their goal :). $sprb"
X Link @RNAiAnalyst 2025-10-06T21:30Z 20.5K followers, 3486 engagements
"$srpt looks like next Saturday there could be a pretty important poster on sirolimus use with elevidys at WMS25:"
X Link @RNAiAnalyst 2025-10-01T21:29Z 20.5K followers, 15.3K engagements
"$qure Sunday for first scientific AMT-130 pivotal trial presentation"
X Link @RNAiAnalyst 2025-10-10T19:33Z 20.5K followers, 24.9K engagements
"$ntla this Biocryst acquisition of $atxs made my day. First claimimf that patients CLEARLY prefer oral for prophylaxis now saying 'sorry we lied to you: every X months and less often much better'. Clearly freaking out about the coming one-time cure. $bcrx"
X Link @RNAiAnalyst 2025-10-14T14:19Z 20.5K followers, 6952 engagements
"@GeneInvesting 100k in US/EU each. Highly underdiagnosed. Could be more. Partly trolling $ALNY but for their own good. I'm just an internet guy ;)"
X Link @RNAiAnalyst 2025-10-16T15:19Z 20.5K followers, XXX engagements
"$mgx remember this pure-play CRISPR Co (partnered with $IONS) trades XX% BELOW CASH"
X Link @RNAiAnalyst 2025-10-16T15:34Z 20.5K followers, 3423 engagements
"@richtrades100 Of course it is about color $srpt provide about current market dynamics plus progress on successfully implementing sirolimus"
X Link @RNAiAnalyst 2025-10-09T09:30Z 20.5K followers, XXX engagements
"Anybody knows whether 'Dear Leader' got an mRNA or the $nvax protein covid vax $bntx $mrna"
X Link @RNAiAnalyst 2025-10-11T11:06Z 20.5K followers, 5594 engagements
"$srpt adds $200M to cash balance in Q2 alone. Balance now at $850M"
X Link @RNAiAnalyst 2025-08-06T20:21Z 20.5K followers, 38.9K engagements
"$ntla believe the next major re-rating of stock occurs when commercial uptake shows patients/KOLs love that type of medicine"
X Link @RNAiAnalyst 2025-10-14T20:34Z 20.5K followers, 5485 engagements
"$NTLA unlike vutrisiran mNIS+7 improves in majority of ATTR-PN patients after NTLA-2001 one time treatment. Another @NEJM publication in the bag. $alny"
X Link @RNAiAnalyst 2025-09-25T14:25Z 20.5K followers, 3792 engagements
"$pepg stock is held almost exclusively by tutes e.g. RA Capital (32%) Point72 (8%); I seem to be the only tax-paying non-tute 😜. Regardless considering $RNA and $DYN market caps of $6B and $2B respectively would be unfair to see offering at anything less than $300M pre-money. $pepg should have announced data first then do an offering after full trading day. Maybe RA Capital wants to add more and that's why"
X Link @RNAiAnalyst 2025-09-24T21:22Z 20.5K followers, 6352 engagements
"$WVE AATD was never the big money maker for Co. It validated the #RNAediting platform. For most applications XX% editing is plenty. Yet it sold off on $BEAM/repeat admin data when obesity and cardiovascular are the crown jewels. Forget about Huntington's and DMD"
X Link @RNAiAnalyst 2025-10-09T20:53Z 20.5K followers, 3535 engagements
"$qure IMO the coming intrathecal RNAi competition need to show superiority to one-time AMT-130. Life-long progressive disease. $alny $arwr"
X Link @RNAiAnalyst 2025-09-26T08:01Z 20.5K followers, 4052 engagements
"💪week: $RNA (also M&A rumors) $DYN (pin action+WMS) $PEPG $SRPT (WMS25). $qure AMT-130 data presentation"
X Link @RNAiAnalyst 2025-10-05T08:41Z 20.5K followers, 4502 engagements
"$SNY be it atopic dermatitis hidradenitis suppuritiva etc.p2 -- p3 results measuring improvement from baseline can differ greatly depending on patient pop (severe/moderate) geography (SOC) etc $NKTR $MTLX"
X Link @RNAiAnalyst 2025-09-04T12:47Z 20.5K followers, 2292 engagements
"$ALNY $REGN: The POWER of #genetic medicine illustrated: (complement C5) mono RNAi better than RNAi-mAb combo despite XX% vs XX% complement assay inhibition respectively for myasthenia gravis. -- why I also expect $NTLA's ATTR and HAE treatment to kick ass. $SLN"
X Link @RNAiAnalyst 2025-08-26T13:28Z 20.5K followers, 6110 engagements
"A backup site What a revolutionary idea. $regn $srrk"
X Link @RNAiAnalyst 2025-10-08T09:38Z 20.5K followers, 4011 engagements
"$abvx in presentation CEO commended team on keeping placebo response low (by limiting enrolment in certain geographies etc) pointing to other recent examples i.e. $mltx and $sny. We all know these'failed' drugs are all active; most of the game is about pbo response mgmt"
X Link @RNAiAnalyst 2025-10-15T14:45Z 20.5K followers, 6554 engagements
"Seems like all the #biotech M&A's get preannounced by the FT or WSJ on Sundays now. Suddenly regret that I was not more aggressive on $RNA in after-hours. My bids did not get filled at $47.8"
X Link @RNAiAnalyst 2025-10-04T13:09Z 20.5K followers, 5598 engagements
"@minimus0987 @SuperDuperDrugs @sonichedgefund @grok CEO said X weeks ago that FDA already saw and reviewed the 240mg dose expansion data incl safety and knockdown and greenlighted 400mg. $wve"
X Link @RNAiAnalyst 2025-10-10T14:23Z 20.5K followers, XXX engagements
"$srpt 2027 convertible debt burden down from $1.2B to $450M with remainder largerly deferred to 2030: Seems like bankruptcy a highly unlikely scenario now and full steam ahead with #RNAi pipeline. $arwr"
X Link @RNAiAnalyst 2025-08-21T11:18Z 20.5K followers, 11.3K engagements
"$srrk $regn smart resourceful traders probably have a list with upcoming PDUFAs for new formulations that rely on the Catalent site. Can I see this list Catalent insiders probably shorting all of them now"
X Link @RNAiAnalyst 2025-10-13T16:55Z 20.5K followers, 6552 engagements
"$10B.that is the combined market cap of major DM1 players $RNA $DYN and $PEPG; add to this $VRTX w/ a approach and stage similar to $PEPG. $RNA also prices in large FSHD opportunity. DM1 is one of the largest genetic disease categories larger than CF. Combined market cap could ultimately be $50B in 2029. There is also likely a succession of therapies coming onto market that improve on earlier approved drugs: 1) $RNA: most advanced evidence of fct improvements but probably least effective on molecular level (CASI) 2) $DYN: a year behind $RNA but CASI superior and impressive fct data at WMS25"
X Link @RNAiAnalyst 2025-10-16T08:42Z 20.5K followers, 4509 engagements
"$prqr I will jinx it but the only explanation for not hearing anything from the company would be M&A. Anything else would be reason for panic. Will have to see whether $nvo jas some leftovers in poxkets after today's $akro acquisition"
X Link @RNAiAnalyst 2025-10-09T19:49Z 20.5K followers, 3646 engagements
"$BIIB $IONS CRL issued for another SMA drug today high-dose nusinersen; and once again it is because of CMC"
X Link @RNAiAnalyst 2025-09-23T20:47Z 20.5K followers, 5573 engagements
"$RNA moving after-hours. Acquisition to be announced ahead of WMS25 Speculating is OK no"
X Link @RNAiAnalyst 2025-10-03T20:10Z 20.5K followers, 4930 engagements
"Down-to-earth data-based discussion of $pepg's DM1 program safety and efficacy. $dyn $rna"
X Link @RNAiAnalyst 2025-10-15T22:27Z 20.5K followers, 8240 engagements
"@Zappitelli1 No way. I'd be terrified having a significant long position going into earnings. Sandy has strung along shareholders quarter after quarter.and he'll continue to do so likely with another highly dilutive financing. $sgmo"
X Link @RNAiAnalyst 2025-08-05T17:59Z 20.5K followers, 1204 engagements
"@anna51296106 $srpt is now studying sirolimus in non-ambulatory. My guess is that if safety positive then back on label h2 2026. $srpt"
X Link @RNAiAnalyst 2025-10-11T12:22Z 20.5K followers, 1580 engagements
"$SLN may be the first RNAi casualty due to competition from China. This Co has done X over last decade in terms of progressing their tech incl extrahepatic delivery as more+more partnering competition for Lpa complement etc emerges"
X Link @RNAiAnalyst 2025-08-29T14:34Z 20.5K followers, 2378 engagements
"$qure Seems that AMT-130 availability could be less than demand initially but I expect that in profit-driven healthcare system hospitals will recognize potential of $clpt-enabled procedures and invest accordingly. 'The most important part of the surgery length beyond people occupying an extremely rare intraoperative MRI scanner are the people who have to be in that room.'"
X Link @RNAiAnalyst 2025-10-05T10:16Z 20.5K followers, 9374 engagements
"$srpt no sirolimus PROPHYLAXIS X of XX with acute liver injury. Sirolimus prophylaxis: zero of X. Proph well tolerated. Single center experience"
X Link @RNAiAnalyst 2025-10-08T12:01Z 20.5K followers, 12.3K engagements
"This guy nailed it. FILL-FINISH.I remember @JMaraganore X decades ago stressing that work not done until fill-finish secured. The irony is that Askshay Vaishnaw went from $ALNY to $SRRK"
X Link @RNAiAnalyst 2025-10-13T13:47Z 20.5K followers, 6418 engagements
"While I see $pepg leading in terms of likely muscle benefits in DM1 remains to be seen how non-muscle symptoms with this cationic peptide conjugate improve relative to the tfr1-targeted antibody conjugates of $rna and $dyne. Similar with $arwr $srpt DM1"
X Link @RNAiAnalyst 2025-10-08T09:08Z 20.5K followers, 3280 engagements
"Decision was last week @statnews reports this morning after new bullish Elevidys data out. Mmh. $srpt"
X Link @RNAiAnalyst 2025-10-08T11:13Z 20.5K followers, 12.7K engagements
"Incredible (and timely) summary on critical importance of EXON1 in #Huntington's disease by unsung heroine Gillian Bates: -- in mouse models XX% exon 1-only kd much more impactful on pathopysiology than XX% full-length kd and much more: $qure $ions $wve $srpt $arwr"
X Link @RNAiAnalyst 2025-10-12T11:15Z 20.5K followers, 9845 engagements
"$mgx moving"
X Link @RNAiAnalyst 2025-10-16T15:32Z 20.5K followers, 2500 engagements
"There is a slide that 'based on current assumptions' there would be a genome editing clinical candidate this year $ions $mgx"
X Link @RNAiAnalyst 2025-10-07T13:09Z 20.5K followers, 1829 engagements
"$srpt getting more and more bullish ahead of earnings: 1) no more deaths since ambulatory only 2) sirolimus as backup (will have to see whether prophylaxis or on-demand wins out) 3) current market would support a $srpt pipeline even without Elevidys"
X Link @RNAiAnalyst 2025-10-08T18:21Z 20.5K followers, 11.7K engagements
"$qure after the initial euphoria and acknowledging the credibility of KOL Tabrizi and Wild can sense a heated bull-bear battle bubbling up. Think $srpt Elevidys. There are vested interests that dont like AMT-130. I also predict that Adam F will become their spokesperson"
X Link @RNAiAnalyst 2025-10-11T22:36Z 20.5K followers, 6614 engagements
"Small #CRISPR platform Co (see also $MGX) Mammoth Biosciences showcases ApoC3 disruption data in NHPs. XX% ApoC3 lowering. Wants to enter clinic for FCS in 2026 to compete with $IONS $ARWR. Looks like nuclease-based. I would recommend base or prime also for gene inhibition"
X Link @RNAiAnalyst 2025-10-09T20:50Z 20.5K followers, 3339 engagements
"@Anonym64382296 @JMaraganore Not on the yahoo feed nor an SEC filing. Had to find this on their website: $SRRK"
X Link @RNAiAnalyst 2025-10-13T14:02Z 20.5K followers, XXX engagements
"#SMA there is no apparent disadvantage from treating CNS only after intrathecal ASO (nusinersen) admin vs systemic small molecule (risdiplam). Very comprehensive analysis. $ions $biib $ptct"
X Link @RNAiAnalyst 2025-10-08T10:28Z 20.5K followers, 2643 engagements
/creator/twitter::RNAiAnalyst