#  @Hall8Jack Jack Hall Jack Hall posts on X about $srpt, $arwr, $rna, $rhhby the most. They currently have [-------] followers and [---] posts still getting attention that total [---------] engagements in the last [--] hours. ### Engagements: [---------] [#](/creator/twitter::2598246906/interactions)  - [--] Week [---------] +88,649% - [--] Month [---------] +44,162% - [--] Months [---------] +8,743% - [--] Year [---------] +3,835% ### Mentions: [--] [#](/creator/twitter::2598246906/posts_active)  - [--] Week [--] -40% - [--] Month [--] +119% - [--] Months [---] +96% - [--] Year [---] +43% ### Followers: [-------] [#](/creator/twitter::2598246906/followers)  - [--] Week [-----] -0.19% - [--] Month [-----] -0.50% - [--] Months [-----] +4.10% - [--] Year [-----] +9.50% ### CreatorRank: [---------] [#](/creator/twitter::2598246906/influencer_rank)  ### Social Influence **Social category influence** [stocks](/list/stocks) [finance](/list/finance) [countries](/list/countries) [cryptocurrencies](/list/cryptocurrencies) [technology brands](/list/technology-brands) [currencies](/list/currencies) [celebrities](/list/celebrities) [fashion brands](/list/fashion-brands) [travel destinations](/list/travel-destinations) [social networks](/list/social-networks) **Social topic influence** [$srpt](/topic/$srpt) #10, [$arwr](/topic/$arwr) #7, [$rna](/topic/$rna), [$rhhby](/topic/$rhhby) #3, [$nvs](/topic/$nvs), [$dyn](/topic/$dyn), [$pepg](/topic/$pepg) #24, [$alny](/topic/$alny), [$pfe](/topic/$pfe), [imo](/topic/imo) **Top accounts mentioned or mentioned by** [@rnaianalyst](/creator/undefined) [@ttramyloid](/creator/undefined) [@gantosj](/creator/undefined) [@semodough](/creator/undefined) [@sportsbios](/creator/undefined) [@sanctuarybio](/creator/undefined) [@yachmod](/creator/undefined) [@bionerd51](/creator/undefined) [@jonu235](/creator/undefined) [@mauriceontw](/creator/undefined) [@buylowsellhighh](/creator/undefined) [@jamesmclaw70633](/creator/undefined) [@biohazard3737](/creator/undefined) [@biotechanalysst](/creator/undefined) [@bikepeddle](/creator/undefined) [@adamfeuerstein](/creator/undefined) [@jfais20](/creator/undefined) [@bikerieder](/creator/undefined) [@sarepta](/creator/undefined) [@pharmdca](/creator/undefined) **Top assets mentioned** [Sarepta Therapeutics, Inc. (SRPT)](/topic/$srpt) [Arrowhead Research Corporation (ARWR)](/topic/$arwr) [Avidity Biosciences, Inc. (RNA)](/topic/$rna) [Novartis AG (NVS)](/topic/$nvs) [Dyne Therapeutics, Inc. (DYN)](/topic/$dyn) [PepGen Inc. (PEPG)](/topic/$pepg) [Alnylam Pharmaceuticals, Inc. (ALNY)](/topic/$alny) [Pfizer, Inc. (PFE)](/topic/$pfe) [uniQure N.V. (QURE)](/topic/$qure) [Wave Life Sciences Ltd. Ordinary Shares (WVE)](/topic/$wve) [Ionis Pharmaceuticals, Inc. Common Stock (IONS)](/topic/$ions) [CRISPR Therapeutics AG (CRSP)](/topic/$crsp) [Eli Lilly and Company (LLY)](/topic/$lly) [Intra-Cellular Therapies Inc. (ITCI)](/topic/$itci) [Viking Therapeutics, Inc (VKTX)](/topic/$vktx) [Vertex Protocol (VRTX)](/topic/$vrtx) [BioMarin Pharmaceutical, Inc. (BMRN)](/topic/$bmrn) [Blueprint Medicines Corporation (BPMC)](/topic/$bpmc) [Summit Therapeutics Inc. Common Stock (SMMT)](/topic/$smmt) [Madrigal Pharmaceuticals, Inc. Common Stock (MDGL)](/topic/$mdgl) [Incyte Corporation (INCY)](/topic/$incy) [Axsome Therapeutics, Inc (AXSM)](/topic/$axsm) [Cytokinetics Inc. (CYTK)](/topic/$cytk) [BridgeBio Pharma, Inc. Common Stock (BBIO)](/topic/$bbio) [Taysha Gene Therapies, Inc. (TSHA)](/topic/$tsha) [NVIDIA Corp. (NVDA)](/topic/$nvda) [Revolution Medicines, Inc. (RVMD)](/topic/$rvmd) [Merck & Co., Inc. (MRK)](/topic/$mrk) [PTC Therapeutics, Inc. (PTCT)](/topic/$ptct) [Neurocrine Biosciences, Inc. (NBIX)](/topic/$nbix) [Insmed, Inc. (INSM)](/topic/$insm) [Bristol-Myers Squibb Co (BMY)](/topic/$bmy) [Abivax SA (ABVX)](/topic/$abvx) [Entrada Therapeutics, Inc. (TRDA)](/topic/$trda) [Moderna Inc (MRNA)](/topic/$mrna) [Exelixis Inc (EXEL)](/topic/$exel) [Krystal Biotech, Inc. Common Stock (KRYS)](/topic/$krys) ### Top Social Posts Top posts by engagements in the last [--] hours "Crazy times $XRH0 $PALL $GLD $PPLT" [X Link](https://x.com/Hall8Jack/status/1217996044727988226) 2020-01-17T02:24Z [----] followers, [--] engagements "Normal behavior $xrh0 $pall" [X Link](https://x.com/Hall8Jack/status/1219285854050553856) 2020-01-20T15:49Z [----] followers, [--] engagements "@NatalieGrover @RxRegA You do realize Unger was moved out of ODE-1 by FDA right after authoring this letter. Why do you suppose they did that" [X Link](https://x.com/Hall8Jack/status/1219938790468399104) 2020-01-22T11:04Z [----] followers, [--] engagements "@NatalieGrover @RxRegA It can be argued both ways: Those that approved were showing substantial bias. Or the individual who wrote CRL was showing substantial bias. To each their own" [X Link](https://x.com/Hall8Jack/status/1219939745230467072) 2020-01-22T11:07Z [----] followers, [--] engagements "@Sports_bios Imo $SRPT will have the clearly superior data but $PFE has a former FDA commish. So I will characterize the competition between the two as a wash" [X Link](https://x.com/Hall8Jack/status/1650177119085903880) 2023-04-23T16:37Z [----] followers, [---] engagements "See @MauriceOnTW $srpt $HNSA :" [X Link](https://x.com/Hall8Jack/status/1754660799463735507) 2024-02-06T00:18Z [----] followers, [----] engagements "@health_stocks What new data This straight from the $RGNX GTx data PR on 2/7 : The Company also expects to share initial strength and functional assessment data for both dose levels and the initiation of a pivotal trial in the second half of [----]. $srpt https://www.prnewswire.com/news-releases/regenxbio-announces-completion-of-enrollment-in-cohort-2-and-additional-positive-interim-data-in-affinity-duchenne-trial-302055713.html" [X Link](https://x.com/Hall8Jack/status/1763612971085758869) 2024-03-01T17:11Z [----] followers, [---] engagements "@RNAiAnalyst I agree - fascinating $srpt $hnsa trial. So what are the key endpoints to know Q225 if successful 1) rh74 Ab titer levels which dictates what % of boys get Elevidys 2) D expression at [--] weeks 3) ongoing safety If so these results will be very interesting" [X Link](https://x.com/Hall8Jack/status/1834987120831701423) 2024-09-14T16:06Z [----] followers, [---] engagements "@RNAiAnalyst And correct me if Im wrong- you dont need to see 100% of patients receiving Elevidys Even 50% of pts SAFELY receiving Elevidys with robust expression would be a huge $hnsa $srpt win" [X Link](https://x.com/Hall8Jack/status/1834988568130764927) 2024-09-14T16:12Z [----] followers, [---] engagements "@MagBiotech Thats a great question Mendell had a track record of success (Zolgensma) and decades of experience with DMD. What is/ will be RegenxBios track record of success #MPS $rgnx $sldb $srpt" [X Link](https://x.com/Hall8Jack/status/1851608376582508787) 2024-10-30T12:53Z [----] followers, [----] engagements "@MagBiotech And look at what $sgmo s potential AA path for Fabry did for its mc recently. Beside DMD could MPS2 have same potential for $rgnx And who might they partner with" [X Link](https://x.com/Hall8Jack/status/1851615671630651487) 2024-10-30T13:22Z [----] followers, [---] engagements "@MagBiotech The market is giving $srpt credit for $Bs in Elevidys revenue; and credit for next to no revenue for rgnx and sldb. So I respectfully disagree. This deal for arwr siRNA is about replacing srpts PMO franchise that is doing $1B per year but they had no pipeline answer- til now" [X Link](https://x.com/Hall8Jack/status/1861402100380340543) 2024-11-26T13:30Z [----] followers, [---] engagements "@ThomasBenderCFA Before today $srpt 3x next year revenue or 5x this year (minus cash). Overvalued 🤔 Really should take a look at $alny $argx $vrtx etc etc etc. 10x next year revenue" [X Link](https://x.com/Hall8Jack/status/1861548231840121073) 2024-11-26T23:10Z [----] followers, [---] engagements "@TTRAmyloid Would not do a deal Cmon this is clearly part of what went on : $arwr $rna $srpt . Oh and $dyn just had a similar clinical hold on similar compound. https://www.prnewswire.com/news-releases/avidity-biosciences-announces-fda-removed-partial-clinical-hold-on-delpacibart-etedesiran-del-desiranaoc-1001-302266275.html https://www.prnewswire.com/news-releases/avidity-biosciences-announces-fda-removed-partial-clinical-hold-on-delpacibart-etedesiran-del-desiranaoc-1001-302266275.html" [X Link](https://x.com/Hall8Jack/status/1862136758890045698) 2024-11-28T14:09Z [----] followers, [----] engagements "@TTRAmyloid Antibody conjugated oligo tox $dyn $rna $arwr $srpt : https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-fda-clinical-hold-ind-application https://www.fiercebiotech.com/biotech/three-execs-resign-dyne-posts-mixed-data-dmd-candidate https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-fda-clinical-hold-ind-application https://www.fiercebiotech.com/biotech/three-execs-resign-dyne-posts-mixed-data-dmd-candidate" [X Link](https://x.com/Hall8Jack/status/1862141964277215366) 2024-11-28T14:30Z [----] followers, [---] engagements "@crisprtalk @semodough Yes a win win- $arwr can be a CV/Metabolics siRNA co and $srpt can be Muscle/CNS siRNA co. ARWR likely can not do both (and then there is Lung). Question is will TRiM work for NM/CNS or will srpt have to develop ligands internally" [X Link](https://x.com/Hall8Jack/status/1863230300555256150) 2024-12-01T14:34Z [----] followers, [---] engagements "@crisprtalk @semodough So beside $arwr partnership -looks like $srpt is now internally a Gene Therapy and siRNA company: .contd https://www.scientistlive.com/content/genetic-disease-trials-sarepta-and-arrowhead https://www.scientistlive.com/content/genetic-disease-trials-sarepta-and-arrowhead" [X Link](https://x.com/Hall8Jack/status/1863231628941668780) 2024-12-01T14:40Z [----] followers, [---] engagements "@crisprtalk @semodough .Sarepta also has license to Arrowheads technology to develop therapeutics against skeletal muscle gene targets. Sareptas Precision Genetic Medicine Engine will also assist in the development of gene therapy RNA technologiesand gene editing. $srpt $arwr" [X Link](https://x.com/Hall8Jack/status/1863232252223635758) 2024-12-01T14:42Z [----] followers, [---] engagements "@crisprtalk @semodough And my guess is $srpt has ALL they can handle with neuromuscular (MuscularDystrophies) - DMD LGMD MD1 FSHD1. That focus alone could get Srpt to $alny valuation" [X Link](https://x.com/Hall8Jack/status/1863263032253505872) 2024-12-01T16:44Z [----] followers, [---] engagements "@semodough Now with a much bigger pipeline $srpt should slowly close some of that huge gap with $vrtx / $alny (8 vs 25/31). They have [--] prominent shots on goal in terms of clinical data in next 12-18 months to try to convince market" [X Link](https://x.com/Hall8Jack/status/1863338562701168684) 2024-12-01T21:45Z [----] followers, [---] engagements "Looks like $rhhby $srpt Elevidys was just approved in Brazil (ages 4-7). For reference there are roughly 10k DMD patients in Brazil; [---] new cases per year: https://www.gov.br/anvisa/pt-br/assuntos/noticias-anvisa/2024/anvisa-aprova-registro-de-primeiro-produto-de-terapia-genica-para-distrofia-muscular-de-duchenne-dmd https://www.gov.br/anvisa/pt-br/assuntos/noticias-anvisa/2024/anvisa-aprova-registro-de-primeiro-produto-de-terapia-genica-para-distrofia-muscular-de-duchenne-dmd" [X Link](https://x.com/Hall8Jack/status/1863686365717635507) 2024-12-02T20:47Z [----] followers, [----] engagements "$srpt $rhhby" [X Link](https://x.com/Hall8Jack/status/1863687846915146213) 2024-12-02T20:53Z [----] followers, [--] engagements "@paulBIOgeek $vrtx has programs in DMD and MD1 (similar to $srpt ) but no news on DMD progress in years and imo $arwr tech (siRNA) is superior to Vertex/Entrada tech (Ab-oligos) in MD1. So imo yes but can they afford" [X Link](https://x.com/Hall8Jack/status/1864377520960733336) 2024-12-04T18:33Z [----] followers, [---] engagements "@paulBIOgeek If $vrtx were smart they would try to sell Exonics to $srpt for $500M ;-) Sarepta if interested could combine Exonics with their Duke IP and Gene Editing Center in NC" [X Link](https://x.com/Hall8Jack/status/1864380681742135599) 2024-12-04T18:46Z [----] followers, [---] engagements "@wkissel $arwr gave $srpt discovery CNS targets so chance they already have licensed out [--] or [--] of those AD proteins:" [X Link](https://x.com/Hall8Jack/status/1864423363734585473) 2024-12-04T21:35Z [----] followers, [----] engagements "@STL_Biotech DMD $srpt and $pfe needed to do PBO trials but for some of these even smaller pop rare dx- PBO trial would be very difficult. The standard of cure is kind of fanciful as very high bar; BP/biotech has never been too interested in that bus model. Academia might need to step in more" [X Link](https://x.com/Hall8Jack/status/1866487218988310592) 2024-12-10T14:16Z [----] followers, [---] engagements "@MelvinRiskMgmt @semodough The ceo has claimed in recent CC peak sales (27) of $5B. Thats 6x GS best case scenario. I think most shareholders would take [---]. lol which would be 5/4x peak sales if they reach $4/5B. Analysts at $3B [--]. $srpt" [X Link](https://x.com/Hall8Jack/status/1869157218421227902) 2024-12-17T23:06Z [----] followers, [---] engagements "@MelvinRiskMgmt @semodough And jeez if you are so offended by $srpt PT what do you think of current $alny and $argx valuations (mind you not PTs) of 12x [--] sales 😡" [X Link](https://x.com/Hall8Jack/status/1869186840772980898) 2024-12-18T01:04Z [----] followers, [---] engagements "@Gantosj And [--] other LGMD Sarcoglycan subtypes following (LGMDc and LGMDd). [--] potential NDAs 2025/2026. $srpt" [X Link](https://x.com/Hall8Jack/status/1869407271371112793) 2024-12-18T15:40Z [----] followers, [---] engagements "$rhhby Brazil agreement on Elevidys $srpt : ➡ @STF_oficial autoriza o cumprimento de decises sobre medicamento Elevidys aps o acordo homologado parcialmente pelo ministro @gilmarmendes entre a Unio e a farmacutica: https://t.co/iwo4RYqdOh #PraTodosVerem: contm descrio da imagem https://t.co/JdDp6yB3lw ➡ @STF_oficial autoriza o cumprimento de decises sobre medicamento Elevidys aps o acordo homologado parcialmente pelo ministro @gilmarmendes entre a Unio e a farmacutica: https://t.co/iwo4RYqdOh #PraTodosVerem: contm descrio da imagem https://t.co/JdDp6yB3lw" [X Link](https://x.com/Hall8Jack/status/1869867328302629049) 2024-12-19T22:08Z [----] followers, [---] engagements "So how much does NS owe $srpt if they in fact owe I think viltepso took in roughly $200M the last [--] years and been on the market [--] years. BREAKING: @Sarepta swayed a federal jury to uphold a patent @NipponShinyaku admitted its Viltepso infringes & to void a patent $SRPT conceded its competing genetic therapy for Duchenne muscular dystrophy infringes. $NPPNY beat willful infringement claims. https://t.co/zRkolVeVOZ BREAKING: @Sarepta swayed a federal jury to uphold a patent @NipponShinyaku admitted its Viltepso infringes & to void a patent $SRPT conceded its competing genetic therapy for" [X Link](https://x.com/Hall8Jack/status/1870155708194586954) 2024-12-20T17:14Z [----] followers, [----] engagements "@buylowsellhighh I am guessing it is. If so then I would assume NS owes $srpt the same profits from Viltepso going forward. If they keep selling it and competing against Vyondys 53" [X Link](https://x.com/Hall8Jack/status/1870225253948875195) 2024-12-20T21:50Z [----] followers, [---] engagements "@yasiejko @Sarepta @uwanews Says in this article : Sarepta voids rivals patent sidestepping $222 million bid. Do you know what this referring to specifically https://news.bloomberglaw.com/ip-law/nippon-shinyaku-owes-sarepta-115-million-on-gene-therapy-patent https://news.bloomberglaw.com/ip-law/nippon-shinyaku-owes-sarepta-115-million-on-gene-therapy-patent" [X Link](https://x.com/Hall8Jack/status/1870266873914503378) 2024-12-21T00:35Z [----] followers, [--] engagements "@TTRAmyloid $rna with clinical hold in DM1 $dyn with clinical hold in DMD Safety is an issue worth watching in these conjugated ASOs" [X Link](https://x.com/Hall8Jack/status/1877750497072791772) 2025-01-10T16:13Z [----] followers, [---] engagements "@auditor112017 @TTRAmyloid What program is this" [X Link](https://x.com/Hall8Jack/status/1877805652388585568) 2025-01-10T19:52Z [----] followers, [--] engagements "@BiotechAnalysst IMO both are attractive assets that will find two different partners with terms vaguely similar to recent $rgnx / NS Pharma deal for MPS GTx: https://finance.yahoo.com/news/regenxbio-nippon-shinyaku-forge-810m-201852428.html https://finance.yahoo.com/news/regenxbio-nippon-shinyaku-forge-810m-201852428.html" [X Link](https://x.com/Hall8Jack/status/1881768273735508448) 2025-01-21T18:18Z [----] followers, [---] engagements "@Jonny_fun_guy @Sports_bios (2/2).the next [--] niche indications imo are sgmo in Fabry and qure in HD. Who will they partner with and will the terms be decent To me these [--] potential partnerships are very key to see how successful GTx smallcos are in [--]. That and can ngne and lxeo file BLAs" [X Link](https://x.com/Hall8Jack/status/1882986737708007776) 2025-01-25T02:59Z [----] followers, [---] engagements "@cognivestment @Pharmdca @MBbiotech @semodough $sldb $srpt Read both of these and ask yourself: who likely played who https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-announces-acquisition-aavantibio-and https://investorrelations.sarepta.com/news-releases/news-release-details/gene-therapy-company-aavantibio-launches-107-million-series https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-announces-acquisition-aavantibio-and" [X Link](https://x.com/Hall8Jack/status/1885876877673328894) 2025-02-02T02:24Z [----] followers, [---] engagements "@Sports_bios The problem is name the companies that could be had at a reasonable premium for $15-30B that could do $3-5B in annual sales Its a Small list I like $alny and $argx but they are too expensive" [X Link](https://x.com/Hall8Jack/status/1886791075924152790) 2025-02-04T14:57Z [----] followers, [---] engagements "@ParaFortuna @Sports_bios Yes but tough to integrate [--] $7B+ deals around same time. I do like the range you mentioned but as Sports pointed out certain companies are going to have to go much larger. IMO more $ITCI type deals ($15B+) coming" [X Link](https://x.com/Hall8Jack/status/1886872491785658571) 2025-02-04T20:20Z [----] followers, [---] engagements "@WallStSai $xbi Larger deals ($10B up) $incy $bntx $nbix $srpt $cytk $bpmc $mdgl $axsm . Others $smmt too expensive/risky imo. $alny $argx too expensive imo" [X Link](https://x.com/Hall8Jack/status/1888638198877651222) 2025-02-09T17:16Z [----] followers, [----] engagements "@jfais20 I like $rgnx but one statement is not very accurate age groups that $srpt cant touch: Sarepta plans on filing BLA expansion for [--] yr olds second half of this year which would imo be well ahead of rgnx:" [X Link](https://x.com/Hall8Jack/status/1889343525558382650) 2025-02-11T15:59Z [----] followers, [----] engagements "@edcollins23 @largelylurking @JonU235 @Nicklaus4Kids Good point wonder if he is confusing with Zolgensma which is AAV9- or another GTx they did clinical trials with. $srpt $nvs" [X Link](https://x.com/Hall8Jack/status/1890373017634500656) 2025-02-14T12:10Z [----] followers, [---] engagements "$rhhby $srpt #Elevidys #Brazil https://www.metropoles.com/colunas/grande-angular/crianca-com-doenca-rara-toma-remedio-de-r-17-mi-quase-na-data-limite https://www.metropoles.com/colunas/grande-angular/crianca-com-doenca-rara-toma-remedio-de-r-17-mi-quase-na-data-limite" [X Link](https://x.com/Hall8Jack/status/1890523651117588494) 2025-02-14T22:08Z [----] followers, [---] engagements "@JonU235 $srpt Another key one for EMA : https://www.mdaconference.org/abstract-library/muscle-mri-outcomes-in-patients-with-duchenne-muscular-dystrophy-treated-with-delandistrogene-moxeparvovec-findings-from-embark-part-1/ https://www.mdaconference.org/abstract-library/muscle-mri-outcomes-in-patients-with-duchenne-muscular-dystrophy-treated-with-delandistrogene-moxeparvovec-findings-from-embark-part-1/" [X Link](https://x.com/Hall8Jack/status/1890632517780213770) 2025-02-15T05:21Z [----] followers, [---] engagements "@SteveHa64191820 @Biotech2k1 $srpt (4x) does appear cheap- at least comparing commercial stage companies he listed based on [----] revenue estimates vs market caps: $bbio (30x) $insm (28x) $axsm (10x) $bpmc (6x)" [X Link](https://x.com/Hall8Jack/status/1891128932067401988) 2025-02-16T14:14Z [----] followers, [--] engagements "@SteveHa64191820 @Biotech2k1 $srpt (4x) does appear cheap- at least comparing commercial stage companies he listed based on [----] revenue estimates vs market caps: $bbio (30x) $insm (28x) $axsm (10x) $bpmc (8x)" [X Link](https://x.com/Hall8Jack/status/1891135908931416204) 2025-02-16T14:41Z [----] followers, [----] engagements "Very key point $srpt $rgnx $sldb : @cognivestment @adamfeuerstein @Pharmdca I would be careful to compare it to Elevidys since Elevidys allows viral antibodies up to 1:400 which is pretty high. Solid Regenx and Genethon are all only dosing if no antibodies (like 1:20 or 1:25). Other than that I think Solid is proving to be safe. @cognivestment @adamfeuerstein @Pharmdca I would be careful to compare it to Elevidys since Elevidys allows viral antibodies up to 1:400 which is pretty high. Solid Regenx and Genethon are all only dosing if no antibodies (like 1:20 or 1:25). Other than that I think" [X Link](https://x.com/Hall8Jack/status/1891983716819304929) 2025-02-18T22:50Z [----] followers, [----] engagements "@BiotechAnalysst This is key $srpt first N=4 mean of 81.2% of muscle fibers expressing micro-dystrophin with a mean intensity of 96% at the sarcolemma. Western blot showed a mean expression of 74.3% without fat or fibrosis adjustment and 95.8% with adjustment. And no SAEs. (1/2)" [X Link](https://x.com/Hall8Jack/status/1892117891614494876) 2025-02-19T07:43Z [----] followers, [---] engagements "@BiotechAnalysst (2/2) This similar to $sldb early N=3. And in $srpt larger N trials for Embark/Endeavor %positive fibers stayed similar to sldb but expression was 34% for Embark. So will expression drop for sldb in larger N also Functional data MOST important(see pfe) http://pubmed.ncbi.nlm.nih.gov/32539076/ http://pubmed.ncbi.nlm.nih.gov/32539076/" [X Link](https://x.com/Hall8Jack/status/1892126229345513574) 2025-02-19T08:17Z [----] followers, [---] engagements "@BiotechAnalysst And obviously need larger N to compare safety properly as again srpt reported no SAEs in first N=4. Altho key is whole field has benefited greatly from pooled $srpt $pfe $sldb Genethon safety data/experience: https://www.fiercebiotech.com/biotech/pfizer-sarepta-team-fellow-dmd-gene-therapy-makers-get-bottom-adverse-events https://www.fiercebiotech.com/biotech/pfizer-sarepta-team-fellow-dmd-gene-therapy-makers-get-bottom-adverse-events" [X Link](https://x.com/Hall8Jack/status/1892126749296644185) 2025-02-19T08:19Z [----] followers, [---] engagements "@Sports_bios (Contd) Its obvious all [--] of these companies pre selected for most ideal patients to raise critical $ at time of initial N=3/4 data. Will come down to much larger trials (N=50 to 100) and will come down to functional data. Jmo" [X Link](https://x.com/Hall8Jack/status/1892272239073964303) 2025-02-19T17:57Z [----] followers, [---] engagements "$rhhby $srpt :" [X Link](https://x.com/Hall8Jack/status/1892411062634369318) 2025-02-20T03:08Z [----] followers, [--] engagements "Wonder if its pfe thats paying stat/AF to dump all over GTx since pfe completely struck out in GTx commercially in embarrassing fashion $nvs $srpt $bmrn $crsp etc: $PFE stops commercialization of hemophilia gene therapy Beqvez that has been approved in April [----] Beqvez is a one-time therapy that was approved in the U.S. last year for the treatment of adults with moderate to severe hemophilia B. $RHHBY $SGMO Beqvez was priced at $3.5 $PFE stops commercialization of hemophilia gene therapy Beqvez that has been approved in April [----] Beqvez is a one-time therapy that was approved in the U.S." [X Link](https://x.com/Hall8Jack/status/1892937524353347950) 2025-02-21T14:00Z [----] followers, [----] engagements "@occams_tooth @PersimmonTI $nvs and Zolgensma just fine too" [X Link](https://x.com/Hall8Jack/status/1893039600869744920) 2025-02-21T20:46Z [----] followers, [--] engagements "@jfais20 $srpt got [--] CNS programs (HD and [--] ataxias) from $arwr in their existing partnership. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-global-licensing-and https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-global-licensing-and" [X Link](https://x.com/Hall8Jack/status/1931007666546954747) 2025-06-06T15:17Z [----] followers, [---] engagements "@jfais20 (Contd) Additionally Sarepta and Arrowhead have entered into a discovery collaboration for up to six additional muscle cardiac and/or CNS targets using Arrowheads novel delivery technologies. $srpt $arwr $rna $dyn $alny" [X Link](https://x.com/Hall8Jack/status/1931007974614384817) 2025-06-06T15:19Z [----] followers, [---] engagements "@LTbioinvestor this data is similar to Elevidys data imo. $rna has bigger market cap than $srpt ; no revenue. And $arwr / $srpt have lower siRNA dose in FSHD" [X Link](https://x.com/Hall8Jack/status/1932080466540347711) 2025-06-09T14:20Z [----] followers, [----] engagements "If reasonable now ask yourself what % of the $arwr pipeline / platform does $srpt own (keeping in mind $rna market cap.) $ARWR is a great example of why public platforms arent getting acquired. They cost too much Capstan set a benchmark with its $2.1B buyout ($500M pre-acqu post money valuation per PB) a 4x step-up on private valuation. Now compare that to $ARWR: 🔹Current market cap: $2B $ARWR is a great example of why public platforms arent getting acquired. They cost too much Capstan set a benchmark with its $2.1B buyout ($500M pre-acqu post money valuation per PB) a 4x step-up on private" [X Link](https://x.com/Hall8Jack/status/1940197699284082739) 2025-07-01T23:55Z [----] followers, [---] engagements "@BowTiedBiotech IMO $rna market cap (first in class muscle targets but likely not best in class) completely backs up this above valuation estimate $arwr $srpt $ARWR (L) TRiM muscle programs use 1/10th - 1/50th the amount of total drug vs $DYN and $RNA Could be benefits from safety standpoint (also potentially allows $SRPT to toggle to quarterly subQ administration vs every 6-8 week IV) Good pace of enrollment data perhaps 2H [--] 🧐 https://t.co/Ava6uXsi6u $ARWR (L) TRiM muscle programs use 1/10th - 1/50th the amount of total drug vs $DYN and $RNA Could be benefits from safety standpoint (also" [X Link](https://x.com/Hall8Jack/status/1940208156057248106) 2025-07-02T00:37Z [----] followers, [---] engagements "Is this one reason why $arwr up big today $200M coming soon from $srpt They expanded their DM1 P1/2 trial to a bigger enrollment than what $rna had in P1/2 and I believe added functional outcomes that rna did not have in P1/2 https://clinicaltrials.gov/study/NCT06138743cond=DM1&term=Arrowhead&rank=1&tab=history&a=9&b=10#version-content-panel https://clinicaltrials.gov/study/NCT06138743cond=DM1&term=Arrowhead&rank=1&tab=history&a=9&b=10#version-content-panel" [X Link](https://x.com/Hall8Jack/status/1955671971322057092) 2025-08-13T16:45Z [----] followers, [---] engagements "(Contd) $rna I believe had DLT (4mg) in their P1/2. So something to compare for $arwr / $srpt in DM1" [X Link](https://x.com/Hall8Jack/status/1955673164807283080) 2025-08-13T16:49Z [----] followers, [---] engagements "@RNAiAnalyst How so Because they accepted $50M in their own stock as payment Or do you think arwr was part of $174M block transaction" [X Link](https://x.com/Hall8Jack/status/1955854576013242755) 2025-08-14T04:50Z [----] followers, [---] engagements "@Amitpat42375370 @blippd @semodough $cytk https://www.royaltypharma.com/news/royalty-pharma-and-cytokinetics-announce-expanded-strategic-funding-collaboration-totaling-up-to-575-million-to-support-commercial-launch-of-aficamten-and-to-advance-rd-pipeline/ https://www.royaltypharma.com/news/royalty-pharma-and-cytokinetics-announce-expanded-strategic-funding-collaboration-totaling-up-to-575-million-to-support-commercial-launch-of-aficamten-and-to-advance-rd-pipeline/" [X Link](https://x.com/Hall8Jack/status/1961817611395571788) 2025-08-30T15:45Z [----] followers, [---] engagements "@Sanctuary_Bio @MauriceOnTW They have early safety signals. Will likely get worse in P3. $sldb $rgnx $srpt" [X Link](https://x.com/Hall8Jack/status/1963299411900960821) 2025-09-03T17:53Z [----] followers, [---] engagements "@yachmod @edcollins23 @MauriceOnTW @Sanctuary_Bio @BiotechObserver May very well still but $srpt took it off their pipeline page. Maybe dont want to step on Roches toes Or they terminated deal Not clear to me. But my guess is gnt0004 has real value as Genethon may be ahead of both $rgnx $sldb. https://www.genethon.com/genethon-to-launch-pivotal-trial-in-europe-of-gnt0004-a-low-dose-microdystrophin-gene-therapy-for-duchenne-muscular-dystrophy/" [X Link](https://x.com/Hall8Jack/status/1963365473892086149) 2025-09-03T22:16Z [----] followers, [---] engagements "@Sanctuary_Bio @BiotechObserver I dont think thats true. Again back to CEOs disclosure problems and keeping in mind the FDA knew about that one death month before all the FDA srpt drama" [X Link](https://x.com/Hall8Jack/status/1963658246117347682) 2025-09-04T17:39Z [----] followers, [---] engagements "@TTRAmyloid But yet $rna insiders dumped $20M the last month. And data is far from slam dunk for stat sig in P3: $arwr $dyn $pepg https://www.aviditybiosciences.com/sites/default/files/2025-04/Final-NMSG-2024-FORTITUDE-Poster-16SEP24.pdf https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf https://www.aviditybiosciences.com/sites/default/files/2025-04/Final-NMSG-2024-FORTITUDE-Poster-16SEP24.pdf" [X Link](https://x.com/Hall8Jack/status/1963745025965355511) 2025-09-04T23:24Z [----] followers, [---] engagements "@TTRAmyloid And $rna DLT (clinical hold) in DM1 is definitely something to watch for vs competitors. https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf" [X Link](https://x.com/Hall8Jack/status/1963746791658037683) 2025-09-04T23:31Z [----] followers, [---] engagements "@TTRAmyloid Has $rna released any functional data on its exon [--] skipper to show that it works (1/2) https://www.prnewswire.com/news-releases/avidity-biosciences-announces-positive-topline-del-zota-data-demonstrating-consistent-statistically-significant-improvements-in-dystrophin-exon-skipping-and-creatine-kinase-in-people-living-with-duchenne-muscular-dystrophy-amena-302402470.html" [X Link](https://x.com/Hall8Jack/status/1963845197474758821) 2025-09-05T06:02Z [----] followers, [---] engagements "@TTRAmyloid (2/2) $rna $srpt $dyn $wve $pepg #NS Exon skip tough from a regulatory standpt in terms of grind it out piecemeal approvals; many skippers have small TAMs (ie exon 44); v crowded in terms of competition. Good summary on DMD exon skipping field/competition: https://musculardystrophynews.com/exon-skipping-for-duchenne-muscular-dystrophy/utm_source=MD&utm_campaign=1f43b91c7c-Email_ENL_US_MD&utm_medium=email&utm_term=0_ea166dad7b-1f43b91c7c-72135529" [X Link](https://x.com/Hall8Jack/status/1963849323847795048) 2025-09-05T06:19Z [----] followers, [---] engagements "@TTRAmyloid To look at actual data $rna Del Zota data seems pretty similar to $srpt Elevidys vs natural history In fact this data favors Elevidys (1/2 contd): Also: https://www.cgtlive.com/view/sarepta-dmd-gene-therapy-elevidys-sustained-benefit-updated-phase-3-data https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-presents-data-american-society-gene-cell https://www.cgtlive.com/view/sarepta-dmd-gene-therapy-elevidys-sustained-benefit-updated-phase-3-data" [X Link](https://x.com/Hall8Jack/status/1965777165636825443) 2025-09-10T13:59Z [----] followers, [----] engagements "@TimCarmichael3 $rna now has discontinuations in [--] of [--] key early phase programs. Not necessarily a platform safety issue but something to keep an eye on.: DMD DM1 https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf https://www.prnewswire.com/news-releases/avidity-biosciences-del-zota-demonstrated-reversal-of-disease-progression-across-key-functional-endpoints-in-explore44-and-explore44-ole-phase-12-trial-in-people-living-with-dmd44-302552339.html" [X Link](https://x.com/Hall8Jack/status/1965890187948679183) 2025-09-10T21:28Z [----] followers, [---] engagements "@scamsweeper (2/2) I believe $arwr has gotten to [--] and [--] mg/kg and $pepg to at least [--] mg/kg . Tiny Pepgen claims better DM1 splicing data than $rna on single dose" [X Link](https://x.com/Hall8Jack/status/1966542196556824781) 2025-09-12T16:39Z [----] followers, [----] engagements "@scamsweeper Compare pepgen splicing data to avidity about same imo: https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA-DMPK-Spliceopathy-Poster-30x42in_V4.0_Final-for-Upload.pdf https://finance.yahoo.com/news/pepgen-announces-positive-initial-results-120000624.html https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA-DMPK-Spliceopathy-Poster-30x42in_V4.0_Final-for-Upload.pdf https://finance.yahoo.com/news/pepgen-announces-positive-initial-results-120000624.html" [X Link](https://x.com/Hall8Jack/status/1966547397376442861) 2025-09-12T17:00Z [----] followers, [---] engagements "This is true with one time GTxs. But key is an overall fatality rate of 1% for $srpt; a one time tx. Juxtapose this vs chronic (lifetime) RNA txs that are seeing discontinuations (and related clinical holds) for $rna $dyn $pepg etc in multiple muscular dystrophy dxs" [X Link](https://x.com/Hall8Jack/status/1970134482629112293) 2025-09-22T14:33Z [----] followers, [----] engagements "@cal89511029 $srpt indicated they would file BLA for Srp-9003 before year end: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-strategic-restructuring-and https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-strategic-restructuring-and" [X Link](https://x.com/Hall8Jack/status/1970544318642541040) 2025-09-23T17:42Z [----] followers, [---] engagements "@blippd @scamsweeper (2/2) so safety is in question for their platform and all comes down to MAD. But imo $pepg has a chance as $rna most advanced in DM1 but efficacy+safety pretty mediocre. Also $arwr DM1 data imminent and also has chance at BIC as think theyve gotten well beyond 4mg/kg" [X Link](https://x.com/Hall8Jack/status/1970958571123982676) 2025-09-24T21:08Z [----] followers, [----] engagements "@blippd @scamsweeper Also only seen splicing data from $pepg If so really need to see DM1 functional data in MAD $rna $dyn $arwr" [X Link](https://x.com/Hall8Jack/status/1970959415273816419) 2025-09-24T21:11Z [----] followers, [---] engagements "@semodough I think $srpt only saw hypoMG in their MAD study for Srp-5051. So the sad study doesnt mean much no $pepg already seen [--] clinical holds. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-update-srp-5051-treatment-duchenne https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-update-srp-5051-treatment-duchenne" [X Link](https://x.com/Hall8Jack/status/1970973689899532693) 2025-09-24T22:08Z [----] followers, [---] engagements "@ShafferBiotech And why no functional data similar to Avidity (which imo was pretty mediocre for efficacy and safety) if splicing data so superior https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf" [X Link](https://x.com/Hall8Jack/status/1970994338181312564) 2025-09-24T23:30Z [----] followers, [---] engagements "@ShafferBiotech $pepg $rna $dyn : $PEPG the functional data at day [--] doesnt seem too different from the $RNA data. Interestingly the functional data only shown upto day [--] for lower [--] doses though based on the study design they should have week [--] biopsy data + other longer term data by now for those doses https://t.co/zc6CFSZQdL $PEPG the functional data at day [--] doesnt seem too different from the $RNA data. Interestingly the functional data only shown upto day [--] for lower [--] doses though based on the study design they should have week [--] biopsy data + other longer term data by now for" [X Link](https://x.com/Hall8Jack/status/1971158467340796192) 2025-09-25T10:22Z [----] followers, [---] engagements "@RNAiAnalyst @JamesMclaw70633 (Contd) see this thread ( $qure $ions $arwr $srpt etc): @Hall8Jack @RNAiAnalyst @JamesMclaw70633 My understanding is that you want to avoid knocking down wild type HTT. Ideally want mutant specific knock-down. As I understand it this was one of the lessons from the failure of Roche / Ionis Tominersen @Hall8Jack @RNAiAnalyst @JamesMclaw70633 My understanding is that you want to avoid knocking down wild type HTT. Ideally want mutant specific knock-down. As I understand it this was one of the lessons from the failure of Roche / Ionis Tominersen" [X Link](https://x.com/Hall8Jack/status/1971516143702872223) 2025-09-26T10:04Z [----] followers, [----] engagements "@RNAiAnalyst Will be tough to compare as the ASOs and siRNA approaches will be running PBO controlled trials and no brain surgery- as opposed to $qure. $ions $alny $arwr $srpt" [X Link](https://x.com/Hall8Jack/status/1971567691849682946) 2025-09-26T13:29Z [----] followers, [---] engagements "@RNAiAnalyst (Contd) so I am sincerely hoping that many HD patients have the legit choice of a COMBO therapy of both gene tx and RNA tx $qure $ions $alny $arwr $srpt etc" [X Link](https://x.com/Hall8Jack/status/1971569804390924553) 2025-09-26T13:37Z [----] followers, [---] engagements "$tsha $ngne #Rett #genetherapy $xbi" [X Link](https://x.com/Hall8Jack/status/1973792844596027801) 2025-10-02T16:51Z [----] followers, [---] engagements "@newerraa And I would guess $arwr / $srpt (Srp-1003) DM1 trial will be updated also in the next several days" [X Link](https://x.com/Hall8Jack/status/1975602595155878281) 2025-10-07T16:42Z [----] followers, [---] engagements "@newerraa Do you have any expectations from the ARO-MMP7 IPF trial $arwr has proven they can get lung exposure with ARO-RAGE but $srpt doesnt mention ARO-MMP7 anymore" [X Link](https://x.com/Hall8Jack/status/1975603842571235671) 2025-10-07T16:47Z [----] followers, [----] engagements "@planert41 IMO eye is on wrong ball. This data matters of course but siRNA data (FSHD/DM1) matters more.again imo $srpt $arwr" [X Link](https://x.com/Hall8Jack/status/1975694009776419248) 2025-10-07T22:45Z [----] followers, [---] engagements "@RNAiAnalyst Did you read this AMT-130 apparently lowers both WT and mutant HTT. Very similar to $arwr / $srpt approach. Obviously $qure intrinsically brain targeted but arwr likely has brain targeting advantages (subQ vs IT) over $ions $alny $wve etc. Should be interesting. I've finally digested the data that Uniqure presented on AMT-130 their gene therapy for Huntington's disease which just announced a positive trial readout with 75% slowing of disease progression. Here is a blog post with my take on the announcement: https://t.co/7Rz1hGQ0X2 I've finally digested the data that Uniqure" [X Link](https://x.com/Hall8Jack/status/1976021537317933451) 2025-10-08T20:27Z [----] followers, [----] engagements "@RNAiAnalyst $qure $arwr :" [X Link](https://x.com/Hall8Jack/status/1976026284993183883) 2025-10-08T20:45Z [----] followers, [---] engagements "@yachmod Think you mean Genethon $srpt used to own US rights to this. Not sure where that stands now: https://www.cgtlive.com/view/genethon-gene-therapy-gnt0004-continues-show-potential-dmd https://www.cgtlive.com/view/genethon-gene-therapy-gnt0004-continues-show-potential-dmd" [X Link](https://x.com/Hall8Jack/status/1976815329713283371) 2025-10-11T01:01Z [----] followers, [---] engagements "@BiotechAnalysst So thats [--] Imlifidase studies from $hnsa - one with Genethon and this from $srpt : Hansa x $SRPT progress. https://t.co/3efOy4Tpoq Hansa x $SRPT progress. https://t.co/3efOy4Tpoq" [X Link](https://x.com/Hall8Jack/status/1978043576035520753) 2025-10-14T10:21Z [----] followers, [----] engagements "$arwr $srpt siRNA IPF trial completed: https://clinicaltrials.gov/study/NCT05537025cond=IPF&term=Arrowhead&rank=1&tab=history&a=23&b=24#version-content-panel https://clinicaltrials.gov/study/NCT05537025cond=IPF&term=Arrowhead&rank=1&tab=history&a=23&b=24#version-content-panel" [X Link](https://x.com/Hall8Jack/status/1978587074350518780) 2025-10-15T22:21Z [----] followers, [----] engagements "@newerraa Interesting $srpt has terminated or withdrawn several trials recently due to business decision (recent restructuring) in LGMDs and Elevidys/plasmapheresis but has not made similar decision in IPF . $arwr " [X Link](https://x.com/Hall8Jack/status/1978669913821643028) 2025-10-16T03:50Z [----] followers, [----] engagements "$xbi $ibb : FDA Commissioner Makary speaking at White House event announces that [--] companies are receiving his Priority Vouchers. More details coming soon but EMD Serono has snagged one. FDA Commissioner Makary speaking at White House event announces that [--] companies are receiving his Priority Vouchers. More details coming soon but EMD Serono has snagged one" [X Link](https://x.com/Hall8Jack/status/1978926320957444457) 2025-10-16T20:49Z [----] followers, [---] engagements "$xbi $ibb : https://www.fda.gov/news-events/press-announcements/fda-awards-first-ever-national-priority-vouchers-nine-sponsors https://www.fda.gov/news-events/press-announcements/fda-awards-first-ever-national-priority-vouchers-nine-sponsors" [X Link](https://x.com/Hall8Jack/status/1978933896457716102) 2025-10-16T21:19Z [----] followers, [---] engagements "@cal89511029 Would this be for LGMD2e gene therapy as has been conjectured $srpt will file bla soon" [X Link](https://x.com/Hall8Jack/status/1980268931211292718) 2025-10-20T13:44Z [----] followers, [---] engagements "@rezfszubagoly @cal89511029 So this might be for a siRNA product (DM1 FSHD IPF) $srpt $arwr" [X Link](https://x.com/Hall8Jack/status/1980279742898303075) 2025-10-20T14:27Z [----] followers, [---] engagements "@Bionerd51 @BruceHarri19541 You claim this based on what precisely $srpt $arwr #ipf" [X Link](https://x.com/Hall8Jack/status/1980675889106170305) 2025-10-21T16:41Z [----] followers, [---] engagements "@suspendedJoey They were supposed to file the HD ARO-HTT IND before the end of the year. Have you seen anything $srpt $arwr" [X Link](https://x.com/Hall8Jack/status/1981336387493200358) 2025-10-23T12:26Z [----] followers, [---] engagements "@Biobeats99 $rhhby $104M this Q (64% growth yoy) 2nd highest since Elevidys exUS launch ( $srpt ) :" [X Link](https://x.com/Hall8Jack/status/1981447144163643660) 2025-10-23T19:46Z [----] followers, [----] engagements "@cal89511029 The 221M chf is for the entire year to date or for [--] Qs. This Q was [---] million chf" [X Link](https://x.com/Hall8Jack/status/1981552070617682227) 2025-10-24T02:43Z [----] followers, [---] engagements "@newerraa The 221M chf is for the entire year to date or for [--] Qs. This Q was [---] million chf" [X Link](https://x.com/Hall8Jack/status/1981552136488935924) 2025-10-24T02:43Z [----] followers, [---] engagements "@RNAiAnalyst @newerraa @Roche @Stocktwits There were shipment stops in Japan (there was a slide on regional Roche sales where it appeared Elevidys had no Japan sales in Q3) Brazil and I think a couple Middle Eastern countries. If so not sure when resume(d). I think current US Elevidys consensus estimate stands at $100M" [X Link](https://x.com/Hall8Jack/status/1981667222465708496) 2025-10-24T10:20Z [----] followers, [---] engagements "@edcollins23 @RNAiAnalyst @newerraa @Roche @Stocktwits Cant argue with you but Roche got to $130M this quarter and Elevidys was/is paused in several of the 9/10 exUS countries where Elevidys was approved. I think US sales have always been greater than exUS. For example- last Q US=$280M; exUS=$90M. $srpt $rhhby" [X Link](https://x.com/Hall8Jack/status/1981724872712532235) 2025-10-24T14:10Z [----] followers, [---] engagements "@PersimmonTI @MichelleF_Davis Why would $rna sell out for like $10B If they get both the DM1 and FSHD markets (ea TAM $1-2B) that should be worth $15-20B market cap. Look at $alny mc of $60B" [X Link](https://x.com/Hall8Jack/status/1982465677886710182) 2025-10-26T15:13Z [----] followers, [---] engagements "Interesting $nvs paid $9B for Zolgensma (Avexis) which does about $1B per year. Now $10B for $rna " [X Link](https://x.com/Hall8Jack/status/1982474051189616686) 2025-10-26T15:47Z [----] followers, [---] engagements "And $rna results are not necessarily unprecedented in any of the indications. $pepg / $dyn showing similar splicing (and functional improvements ) in DM1 and/or FSHD; rna showing similar fold increase (key to compare to baseline) in minidystrophin as other exon skippers for DMD" [X Link](https://x.com/Hall8Jack/status/1982476166146724309) 2025-10-26T15:55Z [----] followers, [---] engagements "@RNAiAnalyst @Gabrielmendesnu Why is $rna DM1 sure thing over FSHD Didnt DM1 have a discontinuation in small early trial Who knows how that manifests itself in much larger P3. $rna $dyn $pepg etc" [X Link](https://x.com/Hall8Jack/status/1982486174082957548) 2025-10-26T16:35Z [----] followers, [---] engagements "@RNAiAnalyst They might be closer for data in FSHD $RNA $dyn $pepg $arwr SRP [----] Update $SRPT https://t.co/sK47i8vak5 SRP [----] Update $SRPT https://t.co/sK47i8vak5" [X Link](https://x.com/Hall8Jack/status/1982514352164164070) 2025-10-26T18:27Z [----] followers, [----] engagements "@commonsenseplay Judging by $nvs / $rna deal imo $srpt s DM1 FSHD [--] Ataxias Huntingtons and IPF $arwr siRNA programs have far far more value than Elevidys " [X Link](https://x.com/Hall8Jack/status/1982520183991443745) 2025-10-26T18:50Z [----] followers, [----] engagements "@PikaCapital (1/3 $rna $dyn $arwr $srpt ) Its a very interesting question if you read this: $ARWR (L) TRiM muscle programs use 1/10th - 1/50th the amount of total drug vs $DYN and $RNA Could be benefits from safety standpoint (also potentially allows $SRPT to toggle to quarterly subQ administration vs every 6-8 week IV) Good pace of enrollment data perhaps 2H [--] 🧐 https://t.co/Ava6uXsi6u $ARWR (L) TRiM muscle programs use 1/10th - 1/50th the amount of total drug vs $DYN and $RNA Could be benefits from safety standpoint (also potentially allows $SRPT to toggle to quarterly subQ administration" [X Link](https://x.com/Hall8Jack/status/1982566441665876023) 2025-10-26T21:54Z [----] followers, [---] engagements "@PikaCapital (2/3 $rna $dyn $arwr $srpt ) or listen to this presentation from RBC: https://kvgo.com/rbc/arrowhead-pharmaceuticals-inc-may-2025 https://kvgo.com/rbc/arrowhead-pharmaceuticals-inc-may-2025" [X Link](https://x.com/Hall8Jack/status/1982566742980501896) 2025-10-26T21:55Z [----] followers, [---] engagements "@PikaCapital (3/3 $rna $dyn $arwr $srpt ) or read this: https://ir.arrowheadpharma.com/static-files/c8100bbd-963d-4275-9233-c0b3f18f6d31 https://ir.arrowheadpharma.com/static-files/c8100bbd-963d-4275-9233-c0b3f18f6d31" [X Link](https://x.com/Hall8Jack/status/1982567190542025184) 2025-10-26T21:57Z [----] followers, [---] engagements "@PikaCapital Key claims via slides $rna $dyn vs $arwr :" [X Link](https://x.com/Hall8Jack/status/1982568677934584139) 2025-10-26T22:03Z [----] followers, [----] engagements "@sonichedgefund Mildly interesting - $nvs PR wording : The Avidity programs feature potential first-in-class late-stage disease-modifying therapies. They dont word it potentially best in class. $rna $dyn $arwr etc" [X Link](https://x.com/Hall8Jack/status/1982595445454246342) 2025-10-26T23:49Z [----] followers, [---] engagements "@DickMedChem Typical $wve - early data hype; not enough meat on the bone" [X Link](https://x.com/Hall8Jack/status/1983634842932932987) 2025-10-29T20:39Z [----] followers, [---] engagements "@financebully @avidresearch Do you think $wve will even be able to raise $ off of this limited data reveal" [X Link](https://x.com/Hall8Jack/status/1983676552236699945) 2025-10-29T23:25Z [----] followers, [---] engagements "@Longroaderstar Looks like $arwr tacking on more market cap gains off of $wve INHBE obesity update than wave is. #ALK7" [X Link](https://x.com/Hall8Jack/status/1983914557404762167) 2025-10-30T15:11Z [----] followers, [---] engagements "@DeuceVault $srpt :" [X Link](https://x.com/Hall8Jack/status/1983992828243145008) 2025-10-30T20:22Z [----] followers, [----] engagements "@BiomedicalRX @PDUFA_Pulse lol $arwr all about INHBE / ALK7 now" [X Link](https://x.com/Hall8Jack/status/1984405357188223023) 2025-10-31T23:41Z [----] followers, [---] engagements "$srpt $arwr : $ARWR adds a couple of arms to its P1/2a myotonic dystrophy study pushes out primary completion date [--] months https://t.co/fM1G9GFRpG $ARWR adds a couple of arms to its P1/2a myotonic dystrophy study pushes out primary completion date [--] months https://t.co/fM1G9GFRpG" [X Link](https://x.com/Hall8Jack/status/1988322907739877734) 2025-11-11T19:08Z [----] followers, [----] engagements "Looks like $arwr $srpt got to subQ for DM1. Discussed at RBC presentation here at [--] min mark: if we can toggle to subQ from IV quarterly then could be transformational vs avidity/dyne. And here: https://kvgo.com/rbc/arrowhead-pharmaceuticals-inc-may-2025 $ARWR (L) TRiM muscle programs use 1/10th - 1/50th the amount of total drug vs $DYN and $RNA Could be benefits from safety standpoint (also potentially allows $SRPT to toggle to quarterly subQ administration vs every 6-8 week IV) Good pace of enrollment data perhaps 2H [--] 🧐 https://t.co/Ava6uXsi6u" [X Link](https://x.com/Hall8Jack/status/1988326972309860815) 2025-11-11T19:24Z [----] followers, [----] engagements "Corrected: at about the [--] min mark; not the [--] min mark for the RBC presentation $arwr $srpt" [X Link](https://x.com/Hall8Jack/status/1988329345572876796) 2025-11-11T19:33Z [----] followers, [---] engagements "Just a reminder avidity P3 DM1 trial IV. . Apparently $arwr $srpt just got to subQ for their P1/2 trial. Could be a very significant differentiator. http://clinicaltrials.gov/study/NCT06411 http://clinicaltrials.gov/study/NCT06411" [X Link](https://x.com/Hall8Jack/status/1988343450635272228) 2025-11-11T20:29Z [----] followers, [---] engagements "@DM1research Avidity/ $nvs hit dose limiting tox at [--] mg/kg I believe for both DM1 and FSHD trials. Whereas $arwr $srpt I think has reached [--] mg/kg cohorts for both DM1/FSHD. And hopefully subQ vs IV" [X Link](https://x.com/Hall8Jack/status/1988358916309123557) 2025-11-11T21:31Z [----] followers, [---] engagements "@veevi24 @DM1research Unfortunately the corresponding Arrowhead trial for FSHD is still only IV arms. But since Arrowhead DM1 trial just added subQ arms it is very possible FSHD will do also since they are similar drugs. https://clinicaltrials.gov/study/NCT06131983cond=FSHD&term=Arrowhead&rank=1 https://clinicaltrials.gov/study/NCT06131983cond=FSHD&term=Arrowhead&rank=1" [X Link](https://x.com/Hall8Jack/status/1988455227562094937) 2025-11-12T03:54Z [----] followers, [--] engagements "@investvee Hoping this is good news for $rhhby $srpt Envol trial and potential approval for boys under [--] who should benefit even more than 4+ ambulatory : https://clinicaltrials.gov/study/NCT06128564cond=DMD&term=Envol&rank=1 https://clinicaltrials.gov/study/NCT06128564cond=DMD&term=Envol&rank=1" [X Link](https://x.com/Hall8Jack/status/1989408124634370542) 2025-11-14T19:00Z [----] followers, [---] engagements "@investvee (Contd) altho have no idea how it affects $srpt BLA submission for SRP-9003 for LGMD2e" [X Link](https://x.com/Hall8Jack/status/1989409201740038457) 2025-11-14T19:04Z [----] followers, [---] engagements "@RNAiAnalyst $wve doesnt even have a DM1 program. $rna $dyn $arwr $pepg $vrtx" [X Link](https://x.com/Hall8Jack/status/1989771432797090157) 2025-11-15T19:04Z [----] followers, [---] engagements "@RNAiAnalyst @JamesMclaw70633 And in terms of potency claim you should listen to $arwr RBC presentation at [--] min mark. Claims very potent and 1/10th to 1/50th of total drug vs $rna $dyn . We shall see https://kvgo.com/rbc/arrowhead-pharmaceuticals-inc-may-2025 https://kvgo.com/rbc/arrowhead-pharmaceuticals-inc-may-2025" [X Link](https://x.com/Hall8Jack/status/1989776331110859052) 2025-11-15T19:23Z [----] followers, [----] engagements "@RNAiAnalyst @JamesMclaw70633 IMO MAD Functional data and safety much more important than SAD splicing data. Hence $pepg only $300m mc $rna $dyn $arwr" [X Link](https://x.com/Hall8Jack/status/1989782687104602593) 2025-11-15T19:48Z [----] followers, [---] engagements "@RNAiAnalyst @JamesMclaw70633 Abs typically 15-50x larger than small protein; FAbs less so. So not that complicated to adjust for relative potency" [X Link](https://x.com/Hall8Jack/status/1989784828074426581) 2025-11-15T19:57Z [----] followers, [--] engagements "@RNAiAnalyst @JamesMclaw70633 Be careful judging from their DMD programs $pepg may have a platform delivery tox issue/disadvantage (kidney/hypoMg) vs $rna . Which is particularly concerning as avidity had a clinical hold in DM1" [X Link](https://x.com/Hall8Jack/status/1989801640283836533) 2025-11-15T21:04Z [----] followers, [---] engagements "@BiotechAnalysst Pull an additional [---] pts from commercial TAM Here says it is an observational study: FDA also required $srpt to run a post-marketing observational study with [---] patients subjected to periodic liver function tests for [--] months after Elevidys tx https://www.biospace.com/fda/fda-imposes-boxed-warning-narrows-patient-pool-for-sareptas-duchenne-gene-therapy-elevidys https://www.biospace.com/fda/fda-imposes-boxed-warning-narrows-patient-pool-for-sareptas-duchenne-gene-therapy-elevidys" [X Link](https://x.com/Hall8Jack/status/1990433313559871950) 2025-11-17T14:54Z [----] followers, [----] engagements "@BiotechAnalysst (Contd) Which i think is Expedition or Endure which is either from commercial pts or pts during a previous clinical study received Elevidys. $srpt https://clinicaltrials.gov/study/NCT05967351cond=DMD&term=Expedition&rank=1 https://clinicaltrials.gov/study/NCT06270719cond=DMD&term=Endure&rank=1 https://clinicaltrials.gov/study/NCT05967351cond=DMD&term=Expedition&rank=1 https://clinicaltrials.gov/study/NCT06270719cond=DMD&term=Endure&rank=1" [X Link](https://x.com/Hall8Jack/status/1990435986283602051) 2025-11-17T15:04Z [----] followers, [---] engagements "@RNAiAnalyst But yet you are betting on $pepg and $dyn over $arwr in DM1. Should be very interesting" [X Link](https://x.com/Hall8Jack/status/1990888409393434659) 2025-11-18T21:02Z [----] followers, [---] engagements "@BikeRieder At [--] min mark - $arwr in FSHD and DM1 we think are great drugs trials progressing well and DM1 should trigger $200M payment from $srpt by end of 2025" [X Link](https://x.com/Hall8Jack/status/1991356252878962880) 2025-11-20T04:01Z [----] followers, [---] engagements "@TrendSpider The day trading trump boys couldnt dump their crypto holdings fast enough lol" [X Link](https://x.com/Hall8Jack/status/1992312837616873837) 2025-11-22T19:22Z [----] followers, [---] engagements "@ArtsStocks If true how did $rna (BO for $12B by $nvs ) $dyn $pepg $vrtx expand into DM1 and/or FSHD w siRNAs and how did $wve expand into INHBE/obesity using siRNAs They have obtained no IP from $ions $alny . All of these companies ( $arwr ) have their own IP based on conjugated delivery" [X Link](https://x.com/Hall8Jack/status/1992999076674375722) 2025-11-24T16:49Z [----] followers, [---] engagements "@ArtsStocks And yes $arwr when they obtained $nvs and $rhhby RNAi assets over [--] years ago had transferred $alny licensing deals which gave arwr freedom to operate" in the RNAi field" [X Link](https://x.com/Hall8Jack/status/1992999825198268726) 2025-11-24T16:52Z [----] followers, [---] engagements "@ArtsStocks So are you saying $nvs wasted their time and $ with $rna BO" [X Link](https://x.com/Hall8Jack/status/1993002899291750783) 2025-11-24T17:04Z [----] followers, [---] engagements "@ArtsStocks Uh huh $nvs blew their DD. IMO you are wildly over interpreting the plozasiran litigation. Which is likely tenuous in and of itself $ions $arwr" [X Link](https://x.com/Hall8Jack/status/1993007014327517456) 2025-11-24T17:21Z [----] followers, [---] engagements "@ArtsStocks You realize the conjugated delivery that $arwr uses for plozasiran is different from the conjugated delivery they use for lung targets; CNS targets and muscle targets; etc. etc. Just curious are you trying to argue $ions has broad claims for delivery to all those targets" [X Link](https://x.com/Hall8Jack/status/1993012600800698853) 2025-11-24T17:43Z [----] followers, [---] engagements "@BioTecIsFuture There are no effective drugs for DM1. Why do you think $nvs BO $rna for $12B Best in class will likely peak north of $2B per year- note these #s based on 10% market share:" [X Link](https://x.com/Hall8Jack/status/1993014422777053479) 2025-11-24T17:50Z [----] followers, [---] engagements "@commonsenseplay Also worth noting avidity ( $nvs BO $12B) hit dose limiting tox at [--] mg/kg in their P1/2 trial. $arwr $srpt" [X Link](https://x.com/Hall8Jack/status/1993048729977557258) 2025-11-24T20:07Z [----] followers, [---] engagements "@commonsenseplay Avidity basically stopped at 4mg/kg. $arwr $srpt now at [--] and [--] mg/kg. https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf" [X Link](https://x.com/Hall8Jack/status/1993049451418820717) 2025-11-24T20:09Z [----] followers, [---] engagements "@commonsenseplay Also $arwr $srpt added subQ cohort to IV cohorts; avidity only IV: https://clinicaltrials.gov/study/NCT06138743cond=DM1&term=Arrowhead&rank=1 https://clinicaltrials.gov/study/NCT06138743cond=DM1&term=Arrowhead&rank=1" [X Link](https://x.com/Hall8Jack/status/1993050412644237758) 2025-11-24T20:13Z [----] followers, [---] engagements "@EasterWing2230 @BiotechAnalysst @yachmod How did the [--] and [--] bonds do today after announcing $200M arwr payment I cant see accurate quotes" [X Link](https://x.com/Hall8Jack/status/1993074266565148810) 2025-11-24T21:48Z [----] followers, [--] engagements "From Dr. Jerry Mendell at NCH #Itvisma / #Zolgensma $nvs . He was also the inventor of $srpt #Elevidys:" [X Link](https://x.com/Hall8Jack/status/1993103350821519825) 2025-11-24T23:44Z [----] followers, [---] engagements "@BowTiedBiotech lol no mention of the debt (ie Celgene) $bmy" [X Link](https://x.com/Hall8Jack/status/1993106427112751233) 2025-11-24T23:56Z [----] followers, [---] engagements "@aakashg0 Interesting theory at same time $nvda / $crwv / OpenAI etc. are deflating we have to now pump $goog AI" [X Link](https://x.com/Hall8Jack/status/1993116269373976707) 2025-11-25T00:35Z [----] followers, [---] engagements "@AIStockSavvy This is worded very poorly. $srpt already said that cohort [--] at 6MPK has started dosing so logically cohort [--] is next at [--] MPK: They also dont mention that $arwr just added new subcutaneous arms in addition to the previous IV arms. Very important detail: https://finance.yahoo.com/news/sarepta-provides-progress-srp-1003-123000296.html https://finance.yahoo.com/news/sarepta-provides-progress-srp-1003-123000296.html" [X Link](https://x.com/Hall8Jack/status/1993329625225674835) 2025-11-25T14:43Z [----] followers, [---] engagements "@AIStockSavvy (Contd) . Here was the original design. So they added a 4.5mpk cohort and then added a subQ arm: . So yes $arwr $srpt made a couple key changes to original design https://ir.arrowheadpharma.com/static-files/c8100bbd-963d-4275-9233-c0b3f18f6d31 https://clinicaltrials.gov/study/NCT06138743cond=DM1&term=Arrowhead&rank=1 https://ir.arrowheadpharma.com/static-files/c8100bbd-963d-4275-9233-c0b3f18f6d31 https://clinicaltrials.gov/study/NCT06138743cond=DM1&term=Arrowhead&rank=1" [X Link](https://x.com/Hall8Jack/status/1993331950560370834) 2025-11-25T14:52Z [----] followers, [---] engagements "@TimCarmichael3 This is a very good point- at this stage of trials $rna (DM1) $dyn (DMD) and $pepg (DMD) all had clinical holds with similar drugs. Not $arwr $srpt in DM1 and FSHD SO FAR" [X Link](https://x.com/Hall8Jack/status/1993360193208418629) 2025-11-25T16:44Z [----] followers, [---] engagements "@TheProfessor_85 $arwr has grown to my largest position but when you look at economics of $srpt deal - if DM1 and/or FSHD hit (ie best in class) - srpt will be worth more than arwr just mo" [X Link](https://x.com/Hall8Jack/status/1993434720982778081) 2025-11-25T21:40Z [----] followers, [---] engagements "@doepke_michel So besides $mdgl - also add $arwr to this potential BO list Or is arwr simply the new spec obesity ticker 🤷🏽" [X Link](https://x.com/Hall8Jack/status/1993702648261095640) 2025-11-26T15:25Z [----] followers, [---] engagements "@ArtsStocks Market certainly doesnt seem as worried about the $ions $arwr litigation risk as you are ;-)" [X Link](https://x.com/Hall8Jack/status/1993707670575145062) 2025-11-26T15:45Z [----] followers, [---] engagements "@BiotechAnalysst @SangamoTx So [--] other interested parties in $rna beside $nvs . Any chance any of them moved onto $arwr or $dyn $srpt etc $XBI how a buyout deal is negotiated: here: $NVS for $RNA RNA contacts [--] third parties [--] of them interested. FYI CC @sangamotx https://t.co/uhXjlvaBsl $XBI how a buyout deal is negotiated: here: $NVS for $RNA RNA contacts [--] third parties [--] of them interested. FYI CC @sangamotx https://t.co/uhXjlvaBsl" [X Link](https://x.com/Hall8Jack/status/1993719721389396401) 2025-11-26T16:33Z [----] followers, [---] engagements "@titteridiot1 @Singlemalt_18 @MorganStanley Very interesting: . Theyve already partnered aro-snca (PD) w $nvs and aro-HTT (HD) w $srpt for CNS. Aro-mapt next In this presentation they seemed most excited about AD: https://ir.arrowheadpharma.com/static-files/c24adf87-2bec-4068-9d55-38da5058c5c9 https://clinicaltrials.gov/study/NCT07221344term=Aro-mapt&rank=1&tab=history https://ir.arrowheadpharma.com/static-files/c24adf87-2bec-4068-9d55-38da5058c5c9 https://clinicaltrials.gov/study/NCT07221344term=Aro-mapt&rank=1&tab=history" [X Link](https://x.com/Hall8Jack/status/1993727207534158229) 2025-11-26T17:03Z [----] followers, [---] engagements "@titteridiot1 @Singlemalt_18 @MorganStanley And looks like $vktx has been dropped for $arwr for obesity spec. Make any sense $wve" [X Link](https://x.com/Hall8Jack/status/1993728162564595846) 2025-11-26T17:06Z [----] followers, [---] engagements "Its all piling up fairly positive for $arwr . FDA validated plozasiran/TRIM. Obesity data soon so $vktx etc spec obesity $ flowing toward arwr. Signed big subQ CNS/PD deal with $nvs . Also have subQ CNS assets in Ataxia; HD ( $srpt ) and AD (unpartnered so additional upfront $ ). Just got $200M payment from srpt for DM1 (and FSHD). Dimer POC Lung target progress Etc etc" [X Link](https://x.com/Hall8Jack/status/1993784415416209627) 2025-11-26T20:50Z [----] followers, [----] engagements "@avidresearch @JacobPlieth $srpt v $capr - apples to oranges but :" [X Link](https://x.com/Hall8Jack/status/1996275806637805578) 2025-12-03T17:50Z [----] followers, [---] engagements "$arwr +12% today presented at PS Conference; worth a listen (also $srpt $nvs ). Was an RNA panel discussion afterward: https://event.webcasts.com/viewer/event.jspei=1742003&tp_key=742b79bc3d https://event.webcasts.com/viewer/event.jspei=1742003&tp_key=742b79bc3d" [X Link](https://x.com/Hall8Jack/status/1996323645468393853) 2025-12-03T21:00Z [----] followers, [----] engagements "$srpt / Endeavor cohort 8/sirolimus related (also $rgnx $sldb etc #GeneTherapy ) : https://musculardystrophynews.com/news/3-drug-cocktail-boosts-dmd-gene-therapy-effectiveness-mice/mc_euid=9b25abc317&utm_source=MD&utm_campaign=a6a5100400-Email_ENL_US_MD&utm_medium=email&utm_term=0_ea166dad7b-a6a5100400-72135529 https://musculardystrophynews.com/news/3-drug-cocktail-boosts-dmd-gene-therapy-effectiveness-mice/mc_euid=9b25abc317&utm_source=MD&utm_campaign=a6a5100400-Email_ENL_US_MD&utm_medium=email&utm_term=0_ea166dad7b-a6a5100400-72135529" [X Link](https://x.com/Hall8Jack/status/1996690038071988399) 2025-12-04T21:16Z [----] followers, [---] engagements "@BikeRieder Little bit surprised $nvs went with $arwr deal for aro-snca/PD instead of aro-mapt/AD. Do you think aro-mapt too expensive asset or nvs saw higher prob of success for aro-snca $Srpt also passed on aro-mapt (in favor of Ataxia1-3; HD CNS assets) so same type of question" [X Link](https://x.com/Hall8Jack/status/1997665895124983933) 2025-12-07T13:53Z [----] followers, [---] engagements "@LSinnv @BikeRieder CNS SC Programs: aro-mapt/AD (arwr) aro-atxn1/Ataxia1 aro-atxn3/Ataxia3 aro-htt/HD (srpt) aro-snca/PD (nvs)" [X Link](https://x.com/Hall8Jack/status/1997745088219496649) 2025-12-07T19:08Z [----] followers, [---] engagements "@richtrades100 @biopharmacaster $dyn safety for x51 looks worse than $srpt . Will be key consideration" [X Link](https://x.com/Hall8Jack/status/1997997837771022847) 2025-12-08T11:53Z [----] followers, [---] engagements "@richtrades100 @biopharmacaster Ppmo-51 had Mg problems. Not Eteplirsen. I stand by my statement. dyn51 had clinical hold. Not good for chronic tx. $dyn $srpt" [X Link](https://x.com/Hall8Jack/status/1997999290875048084) 2025-12-08T11:58Z [----] followers, [---] engagements "$dyn safety not as good as $srpt Etep. Srpt also had ppmo51 which was discontinued due to safety and dyne-251 is very close analog. I would scrutinize ongoing chronic safety data very closely. This safety is very mixed and read note 5- ie why did they choose not to report on safety events after 8/19/25" [X Link](https://x.com/Hall8Jack/status/1998062623040868475) 2025-12-08T16:10Z [----] followers, [----] engagements "@BiotechAnalysst Really need a detailed safety breakdown also" [X Link](https://x.com/Hall8Jack/status/1998103510416519569) 2025-12-08T18:52Z [----] followers, [---] engagements "@BiotechAnalysst No new persistent thrombocytopenia for more than [--] months tough for a chronic tx. $dyn $rna $srpt" [X Link](https://x.com/Hall8Jack/status/1998118972646936878) 2025-12-08T19:54Z [----] followers, [---] engagements "@bioinvestor24 Is this correct summary: $dyn dyne-251 produces 7fold increase of D over baseline $rna del-zota produces 4-5fold increase over baseline And according to this $srpt Etep produces 7fold increase over baseline https://pubmed.ncbi.nlm.nih.gov/34120909/#::text=The%20study%20found:%20*%2078/79%20eteplirsen%2Dtreated%20patientsmild%20to%20moderate%20and%20unrelated%20to%20eteplirsen https://pubmed.ncbi.nlm.nih.gov/34120909/#::text=The%20study%20found:%20*%2078/79%20eteplirsen%2Dtreated%20patientsmild%20to%20moderate%20and%20unrelated%20to%20eteplirsen" [X Link](https://x.com/Hall8Jack/status/1998184443383803973) 2025-12-09T00:14Z [----] followers, [---] engagements "$arwr Related to $srpt subQ CNS Ataxias/Huntingtons Disease programs: https://ir.arrowheadpharma.com/static-files/61bc8b9b-ccf4-45d7-85ad-0d11825bdbc2 https://ir.arrowheadpharma.com/static-files/61bc8b9b-ccf4-45d7-85ad-0d11825bdbc2" [X Link](https://x.com/Hall8Jack/status/1998769645952839969) 2025-12-10T14:59Z [----] followers, [----] engagements "@BikeRieder Do you know if this will be owned by $arwr or this is one of the $srpt agreed to programs" [X Link](https://x.com/Hall8Jack/status/1998787712556605638) 2025-12-10T16:11Z [----] followers, [---] engagements "@FirstSquawk lol Nvda knew full well what was going on" [X Link](https://x.com/Hall8Jack/status/1998819451697758245) 2025-12-10T18:17Z [----] followers, [--] engagements "@BioBoyScout @sonichedgefund @RNAiAnalyst Have to imagine $alny would like to grab $arwr (and the stock is trading like they are in play). But otoh I doubt the FTC would allow the deal to go thru" [X Link](https://x.com/Hall8Jack/status/1999628536361959878) 2025-12-12T23:52Z [----] followers, [---] engagements "@sonichedgefund @BioBoyScout @RNAiAnalyst If on off chance $alny interested in $srpt (I would assume the interest would be around the arwr/srpt muscle; CNS assets) - is the deal with srpt structured such that $arwr can block an acquisition by the likes of alny" [X Link](https://x.com/Hall8Jack/status/1999629616965660790) 2025-12-12T23:57Z [----] followers, [---] engagements "@BioBoyScout I havent noticed them on this schedule yet. Do you know exact day and or timeslot $arwr #jpm The #JPM26 schedule is already out. https://t.co/0ZtclKXxdb The #JPM26 schedule is already out. https://t.co/0ZtclKXxdb" [X Link](https://x.com/Hall8Jack/status/2001038593964347878) 2025-12-16T21:15Z [----] followers, [---] engagements "$capr Interesting point- was said [--] of the trial pts were actively on exon skippers. Also Dr McD said even tho most pts were late/borderline ambulatory they did not test for lower limb function. As such thought approval should be consistent with ages in trial (ie dont repeat the mistake $srpt made). Key point for me- in CRL there were manufacturing/CMC issues. Capr has responded on those issues but nothing back from FDA yet" [X Link](https://x.com/Hall8Jack/status/2001376124216185271) 2025-12-17T19:37Z [----] followers, [---] engagements "@biotech_hunter (Contd). Dr McD said they probably would run trial with younger patients. Seems like a no brainer. Also he seemed to indicate not sure if he would give Deramiocel for entire lifetime of patient but would be comfortable for 5+ years at least. $capr" [X Link](https://x.com/Hall8Jack/status/2001377055209111950) 2025-12-17T19:40Z [----] followers, [---] engagements "@biotech_hunter Could be but I simply heard $capr responded and hoped would be sufficient with FDA. But I heard nothing about FDA specific response back. So an open issue for me. Deramiocel is donor derived and need repeat doses so I imagine scale up and standardization is a tricky issue" [X Link](https://x.com/Hall8Jack/status/2001381572034130096) 2025-12-17T19:58Z [----] followers, [---] engagements "@IroquoiInvest $srpt Thats what HHS official said in Newsweek etc: https://www.newsweek.com/hhs-official-newborn-screening-saves-lives-and-empowers-families-opinion-11217094 https://www.newsweek.com/hhs-official-newborn-screening-saves-lives-and-empowers-families-opinion-11217094" [X Link](https://x.com/Hall8Jack/status/2001752278680690854) 2025-12-18T20:31Z [----] followers, [----] engagements "@IroquoiInvest Also great to see Really strong support for these New Born Screenings. VERY Important See all the comments as you scroll down in What They Are Saying link: https://www.hhs.gov/press-room/wtas-secretary-kennedy-adds-duchenne-muscular-dystrophy-metachromatic-leukodystrophy-to-newborn-screenings.html https://www.hhs.gov/press-room/wtas-secretary-kennedy-adds-duchenne-muscular-dystrophy-metachromatic-leukodystrophy-to-newborn-screenings.html" [X Link](https://x.com/Hall8Jack/status/2001754027776761930) 2025-12-18T20:38Z [----] followers, [--] engagements "@JackFarley96 lol think about the markets caps (and insider liquidation events) and debt of much bigger companies that are riding on this $msft $amzn $meta $nvda $avgo $orcl etc etc. SA is literally banking on this" [X Link](https://x.com/Hall8Jack/status/2001833758232871074) 2025-12-19T01:55Z [----] followers, [----] engagements "@RNAiAnalyst Such an insufferable shill 🤡 . God forbid $lly or some other BP have to pay an extra $10 for $abvx" [X Link](https://x.com/Hall8Jack/status/2006148151888023733) 2025-12-30T23:39Z [----] followers, [----] engagements "@obryanjf At least $srpt progress on Endeavor- recruiting again. Cohort [--] (sirolimus) is new of course. Not sure if good sign for LGMD2e BLA filing https://clinicaltrials.gov/study/NCT04626674cond=DMD&term=Endeavor&rank=1&tab=history&a=11&b=12#version-content-panel https://clinicaltrials.gov/study/NCT04626674cond=DMD&term=Endeavor&rank=1&tab=history&a=11&b=12#version-content-panel" [X Link](https://x.com/Hall8Jack/status/2007159581223567406) 2026-01-02T18:38Z [----] followers, [---] engagements "@RNAiAnalyst According to preclinical data Srp-1005 knocks down both WT and mutant HTT- similar to $qure and $ions approaches. $arwr $srpt" [X Link](https://x.com/Hall8Jack/status/2008947243038953573) 2026-01-07T17:02Z [----] followers, [---] engagements "@BioSignal Co-Development So you think the portfolio company will partner in US with someone like Alnylam or Big Pharma" [X Link](https://x.com/Hall8Jack/status/2009093473161621662) 2026-01-08T02:43Z [----] followers, [--] engagements "@MarkC_LVB @ArrowheadPharma Yep here is the spec that APOE could be the next big $arwr CNS program (after PD AD/MAPT $srpt HD): New on Substack (Free): $ARWR APOE on the Menu: What Arrowheads BBB Patent Actually Tells Us A look at the CNS Target X slide APOEs cardiometabolic/CNS biology and Arrowheads new TfR1 BBB patent. How APOE emerges as a clean prototype for a Target Xstyle program and New on Substack (Free): $ARWR APOE on the Menu: What Arrowheads BBB Patent Actually Tells Us A look at the CNS Target X slide APOEs cardiometabolic/CNS biology and Arrowheads new TfR1 BBB patent. How APOE" [X Link](https://x.com/Hall8Jack/status/2009699253598437642) 2026-01-09T18:50Z [----] followers, [----] engagements "And remember as part of $arwr $srpt deal- entered into a discovery collaboration for up to six additional muscle cardiac and/or CNS targets. Would be nice to hear more about those preclinical siRNA programs at JPM. Have seen some interesting targets in their patents: for ex APOE ACVR2 MSTN GYS1. https://twitter.com/i/web/status/2010040530588103102 https://twitter.com/i/web/status/2010040530588103102" [X Link](https://x.com/Hall8Jack/status/2010040530588103102) 2026-01-10T17:26Z [----] followers, [----] engagements "@RNAiAnalyst You may be focused on the wrong companies: see below: $arwr #APOE #AD New on Substack (Free): $ARWR APOE on the Menu: What Arrowheads BBB Patent Actually Tells Us A look at the CNS Target X slide APOEs cardiometabolic/CNS biology and Arrowheads new TfR1 BBB patent. How APOE emerges as a clean prototype for a Target Xstyle program and New on Substack (Free): $ARWR APOE on the Menu: What Arrowheads BBB Patent Actually Tells Us A look at the CNS Target X slide APOEs cardiometabolic/CNS biology and Arrowheads new TfR1 BBB patent. How APOE emerges as a clean prototype for a Target" [X Link](https://x.com/Hall8Jack/status/2010105640929374282) 2026-01-10T21:45Z [----] followers, [---] engagements "@RNAiAnalyst (Contd) $arwr ( $srpt ) #APOE etc: https://patents.google.com/patent/WO2025160275A1/en https://patents.google.com/patent/WO2025160275A1/en" [X Link](https://x.com/Hall8Jack/status/2010115735650013373) 2026-01-10T22:25Z [----] followers, [---] engagements "@Marktillerimark @tickerade Potential targets as part of $arwr $srpt agreement: @RNAiAnalyst (Contd) $arwr ( $srpt ) #APOE etc: https://t.co/09w5Zuj7jd https://t.co/5QyaYAH15L @RNAiAnalyst (Contd) $arwr ( $srpt ) #APOE etc: https://t.co/09w5Zuj7jd https://t.co/5QyaYAH15L" [X Link](https://x.com/Hall8Jack/status/2010118355240292448) 2026-01-10T22:35Z [----] followers, [---] engagements "@Marktillerimark @tickerade Contd $arwr $srpt :" [X Link](https://x.com/Hall8Jack/status/2010121473969537409) 2026-01-10T22:48Z [----] followers, [---] engagements "@BiotechObserver Not sure how you can say most boring ever when there are credible rumors of $30B and $20B advanced BO negotiations. Rumors and media backed spec yes but not boring imo. $rvmd $abvx $mrk $abbv $lly $jnj $azn $xbi #jpm" [X Link](https://x.com/Hall8Jack/status/2010873247705817176) 2026-01-13T00:35Z [----] followers, [----] engagements "@timfris Would not be shocked if $axsm leap frogs both $rvmd / $abvx . $xbi" [X Link](https://x.com/Hall8Jack/status/2011279681547616478) 2026-01-14T03:30Z [----] followers, [---] engagements "Yes I have seen some complaining boring JPM. Investing doesnt necessarily need drama. Look at big tech the last few years- a ton of slow steady multiple expansion. Versus multiple compression in $XBI for years. But weve had huge breakthrus in cancer obesity rare dx etc genetic medicine etc. And now in midst of boring JPM - credible rumors of $20B and $30B BO talks amidst BP patent cliffs. These are the key factors that lead to ongoing multiple expansion once again. https://twitter.com/i/web/status/2011498392698974560 https://twitter.com/i/web/status/2011498392698974560" [X Link](https://x.com/Hall8Jack/status/2011498392698974560) 2026-01-14T17:59Z [----] followers, [--] engagements "@qian_li41394 Hmm did this list Underperform this week" [X Link](https://x.com/Hall8Jack/status/2012270763047334050) 2026-01-16T21:08Z [----] followers, [--] engagements Limited data mode. Full metrics available with subscription: lunarcrush.com/pricing
@Hall8Jack Jack Hall
Jack Hall posts on X about $srpt, $arwr, $rna, $rhhby the most. They currently have [-------] followers and [---] posts still getting attention that total [---------] engagements in the last [--] hours.
Engagements: [---------] #
- [--] Week [---------] +88,649%
- [--] Month [---------] +44,162%
- [--] Months [---------] +8,743%
- [--] Year [---------] +3,835%
Mentions: [--] #
- [--] Week [--] -40%
- [--] Month [--] +119%
- [--] Months [---] +96%
- [--] Year [---] +43%
Followers: [-------] #
- [--] Week [-----] -0.19%
- [--] Month [-----] -0.50%
- [--] Months [-----] +4.10%
- [--] Year [-----] +9.50%
CreatorRank: [---------] #
Social Influence
Social category influence stocks finance countries cryptocurrencies technology brands currencies celebrities fashion brands travel destinations social networks
Social topic influence $srpt #10, $arwr #7, $rna, $rhhby #3, $nvs, $dyn, $pepg #24, $alny, $pfe, imo
Top accounts mentioned or mentioned by @rnaianalyst @ttramyloid @gantosj @semodough @sportsbios @sanctuarybio @yachmod @bionerd51 @jonu235 @mauriceontw @buylowsellhighh @jamesmclaw70633 @biohazard3737 @biotechanalysst @bikepeddle @adamfeuerstein @jfais20 @bikerieder @sarepta @pharmdca
Top assets mentioned Sarepta Therapeutics, Inc. (SRPT) Arrowhead Research Corporation (ARWR) Avidity Biosciences, Inc. (RNA) Novartis AG (NVS) Dyne Therapeutics, Inc. (DYN) PepGen Inc. (PEPG) Alnylam Pharmaceuticals, Inc. (ALNY) Pfizer, Inc. (PFE) uniQure N.V. (QURE) Wave Life Sciences Ltd. Ordinary Shares (WVE) Ionis Pharmaceuticals, Inc. Common Stock (IONS) CRISPR Therapeutics AG (CRSP) Eli Lilly and Company (LLY) Intra-Cellular Therapies Inc. (ITCI) Viking Therapeutics, Inc (VKTX) Vertex Protocol (VRTX) BioMarin Pharmaceutical, Inc. (BMRN) Blueprint Medicines Corporation (BPMC) Summit Therapeutics Inc. Common Stock (SMMT) Madrigal Pharmaceuticals, Inc. Common Stock (MDGL) Incyte Corporation (INCY) Axsome Therapeutics, Inc (AXSM) Cytokinetics Inc. (CYTK) BridgeBio Pharma, Inc. Common Stock (BBIO) Taysha Gene Therapies, Inc. (TSHA) NVIDIA Corp. (NVDA) Revolution Medicines, Inc. (RVMD) Merck & Co., Inc. (MRK) PTC Therapeutics, Inc. (PTCT) Neurocrine Biosciences, Inc. (NBIX) Insmed, Inc. (INSM) Bristol-Myers Squibb Co (BMY) Abivax SA (ABVX) Entrada Therapeutics, Inc. (TRDA) Moderna Inc (MRNA) Exelixis Inc (EXEL) Krystal Biotech, Inc. Common Stock (KRYS)
Top Social Posts
Top posts by engagements in the last [--] hours
"Crazy times $XRH0 $PALL $GLD $PPLT"
X Link 2020-01-17T02:24Z [----] followers, [--] engagements
"Normal behavior $xrh0 $pall"
X Link 2020-01-20T15:49Z [----] followers, [--] engagements
"@NatalieGrover @RxRegA You do realize Unger was moved out of ODE-1 by FDA right after authoring this letter. Why do you suppose they did that"
X Link 2020-01-22T11:04Z [----] followers, [--] engagements
"@NatalieGrover @RxRegA It can be argued both ways: Those that approved were showing substantial bias. Or the individual who wrote CRL was showing substantial bias. To each their own"
X Link 2020-01-22T11:07Z [----] followers, [--] engagements
"@Sports_bios Imo $SRPT will have the clearly superior data but $PFE has a former FDA commish. So I will characterize the competition between the two as a wash"
X Link 2023-04-23T16:37Z [----] followers, [---] engagements
"See @MauriceOnTW $srpt $HNSA :"
X Link 2024-02-06T00:18Z [----] followers, [----] engagements
"@health_stocks What new data This straight from the $RGNX GTx data PR on 2/7 : The Company also expects to share initial strength and functional assessment data for both dose levels and the initiation of a pivotal trial in the second half of [----]. $srpt https://www.prnewswire.com/news-releases/regenxbio-announces-completion-of-enrollment-in-cohort-2-and-additional-positive-interim-data-in-affinity-duchenne-trial-302055713.html"
X Link 2024-03-01T17:11Z [----] followers, [---] engagements
"@RNAiAnalyst I agree - fascinating $srpt $hnsa trial. So what are the key endpoints to know Q225 if successful 1) rh74 Ab titer levels which dictates what % of boys get Elevidys 2) D expression at [--] weeks 3) ongoing safety If so these results will be very interesting"
X Link 2024-09-14T16:06Z [----] followers, [---] engagements
"@RNAiAnalyst And correct me if Im wrong- you dont need to see 100% of patients receiving Elevidys Even 50% of pts SAFELY receiving Elevidys with robust expression would be a huge $hnsa $srpt win"
X Link 2024-09-14T16:12Z [----] followers, [---] engagements
"@MagBiotech Thats a great question Mendell had a track record of success (Zolgensma) and decades of experience with DMD. What is/ will be RegenxBios track record of success #MPS $rgnx $sldb $srpt"
X Link 2024-10-30T12:53Z [----] followers, [----] engagements
"@MagBiotech And look at what $sgmo s potential AA path for Fabry did for its mc recently. Beside DMD could MPS2 have same potential for $rgnx And who might they partner with"
X Link 2024-10-30T13:22Z [----] followers, [---] engagements
"@MagBiotech The market is giving $srpt credit for $Bs in Elevidys revenue; and credit for next to no revenue for rgnx and sldb. So I respectfully disagree. This deal for arwr siRNA is about replacing srpts PMO franchise that is doing $1B per year but they had no pipeline answer- til now"
X Link 2024-11-26T13:30Z [----] followers, [---] engagements
"@ThomasBenderCFA Before today $srpt 3x next year revenue or 5x this year (minus cash). Overvalued 🤔 Really should take a look at $alny $argx $vrtx etc etc etc. 10x next year revenue"
X Link 2024-11-26T23:10Z [----] followers, [---] engagements
"@TTRAmyloid Would not do a deal Cmon this is clearly part of what went on : $arwr $rna $srpt . Oh and $dyn just had a similar clinical hold on similar compound. https://www.prnewswire.com/news-releases/avidity-biosciences-announces-fda-removed-partial-clinical-hold-on-delpacibart-etedesiran-del-desiranaoc-1001-302266275.html https://www.prnewswire.com/news-releases/avidity-biosciences-announces-fda-removed-partial-clinical-hold-on-delpacibart-etedesiran-del-desiranaoc-1001-302266275.html"
X Link 2024-11-28T14:09Z [----] followers, [----] engagements
"@TTRAmyloid Antibody conjugated oligo tox $dyn $rna $arwr $srpt : https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-fda-clinical-hold-ind-application https://www.fiercebiotech.com/biotech/three-execs-resign-dyne-posts-mixed-data-dmd-candidate https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-fda-clinical-hold-ind-application https://www.fiercebiotech.com/biotech/three-execs-resign-dyne-posts-mixed-data-dmd-candidate"
X Link 2024-11-28T14:30Z [----] followers, [---] engagements
"@crisprtalk @semodough Yes a win win- $arwr can be a CV/Metabolics siRNA co and $srpt can be Muscle/CNS siRNA co. ARWR likely can not do both (and then there is Lung). Question is will TRiM work for NM/CNS or will srpt have to develop ligands internally"
X Link 2024-12-01T14:34Z [----] followers, [---] engagements
"@crisprtalk @semodough So beside $arwr partnership -looks like $srpt is now internally a Gene Therapy and siRNA company: .contd https://www.scientistlive.com/content/genetic-disease-trials-sarepta-and-arrowhead https://www.scientistlive.com/content/genetic-disease-trials-sarepta-and-arrowhead"
X Link 2024-12-01T14:40Z [----] followers, [---] engagements
"@crisprtalk @semodough .Sarepta also has license to Arrowheads technology to develop therapeutics against skeletal muscle gene targets. Sareptas Precision Genetic Medicine Engine will also assist in the development of gene therapy RNA technologiesand gene editing. $srpt $arwr"
X Link 2024-12-01T14:42Z [----] followers, [---] engagements
"@crisprtalk @semodough And my guess is $srpt has ALL they can handle with neuromuscular (MuscularDystrophies) - DMD LGMD MD1 FSHD1. That focus alone could get Srpt to $alny valuation"
X Link 2024-12-01T16:44Z [----] followers, [---] engagements
"@semodough Now with a much bigger pipeline $srpt should slowly close some of that huge gap with $vrtx / $alny (8 vs 25/31). They have [--] prominent shots on goal in terms of clinical data in next 12-18 months to try to convince market"
X Link 2024-12-01T21:45Z [----] followers, [---] engagements
"Looks like $rhhby $srpt Elevidys was just approved in Brazil (ages 4-7). For reference there are roughly 10k DMD patients in Brazil; [---] new cases per year: https://www.gov.br/anvisa/pt-br/assuntos/noticias-anvisa/2024/anvisa-aprova-registro-de-primeiro-produto-de-terapia-genica-para-distrofia-muscular-de-duchenne-dmd https://www.gov.br/anvisa/pt-br/assuntos/noticias-anvisa/2024/anvisa-aprova-registro-de-primeiro-produto-de-terapia-genica-para-distrofia-muscular-de-duchenne-dmd"
X Link 2024-12-02T20:47Z [----] followers, [----] engagements
"$srpt $rhhby"
X Link 2024-12-02T20:53Z [----] followers, [--] engagements
"@paulBIOgeek $vrtx has programs in DMD and MD1 (similar to $srpt ) but no news on DMD progress in years and imo $arwr tech (siRNA) is superior to Vertex/Entrada tech (Ab-oligos) in MD1. So imo yes but can they afford"
X Link 2024-12-04T18:33Z [----] followers, [---] engagements
"@paulBIOgeek If $vrtx were smart they would try to sell Exonics to $srpt for $500M ;-) Sarepta if interested could combine Exonics with their Duke IP and Gene Editing Center in NC"
X Link 2024-12-04T18:46Z [----] followers, [---] engagements
"@wkissel $arwr gave $srpt discovery CNS targets so chance they already have licensed out [--] or [--] of those AD proteins:"
X Link 2024-12-04T21:35Z [----] followers, [----] engagements
"@STL_Biotech DMD $srpt and $pfe needed to do PBO trials but for some of these even smaller pop rare dx- PBO trial would be very difficult. The standard of cure is kind of fanciful as very high bar; BP/biotech has never been too interested in that bus model. Academia might need to step in more"
X Link 2024-12-10T14:16Z [----] followers, [---] engagements
"@MelvinRiskMgmt @semodough The ceo has claimed in recent CC peak sales (27) of $5B. Thats 6x GS best case scenario. I think most shareholders would take [---]. lol which would be 5/4x peak sales if they reach $4/5B. Analysts at $3B [--]. $srpt"
X Link 2024-12-17T23:06Z [----] followers, [---] engagements
"@MelvinRiskMgmt @semodough And jeez if you are so offended by $srpt PT what do you think of current $alny and $argx valuations (mind you not PTs) of 12x [--] sales 😡"
X Link 2024-12-18T01:04Z [----] followers, [---] engagements
"@Gantosj And [--] other LGMD Sarcoglycan subtypes following (LGMDc and LGMDd). [--] potential NDAs 2025/2026. $srpt"
X Link 2024-12-18T15:40Z [----] followers, [---] engagements
"$rhhby Brazil agreement on Elevidys $srpt : ➡ @STF_oficial autoriza o cumprimento de decises sobre medicamento Elevidys aps o acordo homologado parcialmente pelo ministro @gilmarmendes entre a Unio e a farmacutica: https://t.co/iwo4RYqdOh #PraTodosVerem: contm descrio da imagem https://t.co/JdDp6yB3lw ➡ @STF_oficial autoriza o cumprimento de decises sobre medicamento Elevidys aps o acordo homologado parcialmente pelo ministro @gilmarmendes entre a Unio e a farmacutica: https://t.co/iwo4RYqdOh #PraTodosVerem: contm descrio da imagem https://t.co/JdDp6yB3lw"
X Link 2024-12-19T22:08Z [----] followers, [---] engagements
"So how much does NS owe $srpt if they in fact owe I think viltepso took in roughly $200M the last [--] years and been on the market [--] years. BREAKING: @Sarepta swayed a federal jury to uphold a patent @NipponShinyaku admitted its Viltepso infringes & to void a patent $SRPT conceded its competing genetic therapy for Duchenne muscular dystrophy infringes. $NPPNY beat willful infringement claims. https://t.co/zRkolVeVOZ BREAKING: @Sarepta swayed a federal jury to uphold a patent @NipponShinyaku admitted its Viltepso infringes & to void a patent $SRPT conceded its competing genetic therapy for"
X Link 2024-12-20T17:14Z [----] followers, [----] engagements
"@buylowsellhighh I am guessing it is. If so then I would assume NS owes $srpt the same profits from Viltepso going forward. If they keep selling it and competing against Vyondys 53"
X Link 2024-12-20T21:50Z [----] followers, [---] engagements
"@yasiejko @Sarepta @uwanews Says in this article : Sarepta voids rivals patent sidestepping $222 million bid. Do you know what this referring to specifically https://news.bloomberglaw.com/ip-law/nippon-shinyaku-owes-sarepta-115-million-on-gene-therapy-patent https://news.bloomberglaw.com/ip-law/nippon-shinyaku-owes-sarepta-115-million-on-gene-therapy-patent"
X Link 2024-12-21T00:35Z [----] followers, [--] engagements
"@TTRAmyloid $rna with clinical hold in DM1 $dyn with clinical hold in DMD Safety is an issue worth watching in these conjugated ASOs"
X Link 2025-01-10T16:13Z [----] followers, [---] engagements
"@auditor112017 @TTRAmyloid What program is this"
X Link 2025-01-10T19:52Z [----] followers, [--] engagements
"@BiotechAnalysst IMO both are attractive assets that will find two different partners with terms vaguely similar to recent $rgnx / NS Pharma deal for MPS GTx: https://finance.yahoo.com/news/regenxbio-nippon-shinyaku-forge-810m-201852428.html https://finance.yahoo.com/news/regenxbio-nippon-shinyaku-forge-810m-201852428.html"
X Link 2025-01-21T18:18Z [----] followers, [---] engagements
"@Jonny_fun_guy @Sports_bios (2/2).the next [--] niche indications imo are sgmo in Fabry and qure in HD. Who will they partner with and will the terms be decent To me these [--] potential partnerships are very key to see how successful GTx smallcos are in [--]. That and can ngne and lxeo file BLAs"
X Link 2025-01-25T02:59Z [----] followers, [---] engagements
"@cognivestment @Pharmdca @MBbiotech @semodough $sldb $srpt Read both of these and ask yourself: who likely played who https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-announces-acquisition-aavantibio-and https://investorrelations.sarepta.com/news-releases/news-release-details/gene-therapy-company-aavantibio-launches-107-million-series https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-announces-acquisition-aavantibio-and"
X Link 2025-02-02T02:24Z [----] followers, [---] engagements
"@Sports_bios The problem is name the companies that could be had at a reasonable premium for $15-30B that could do $3-5B in annual sales Its a Small list I like $alny and $argx but they are too expensive"
X Link 2025-02-04T14:57Z [----] followers, [---] engagements
"@ParaFortuna @Sports_bios Yes but tough to integrate [--] $7B+ deals around same time. I do like the range you mentioned but as Sports pointed out certain companies are going to have to go much larger. IMO more $ITCI type deals ($15B+) coming"
X Link 2025-02-04T20:20Z [----] followers, [---] engagements
"@WallStSai $xbi Larger deals ($10B up) $incy $bntx $nbix $srpt $cytk $bpmc $mdgl $axsm . Others $smmt too expensive/risky imo. $alny $argx too expensive imo"
X Link 2025-02-09T17:16Z [----] followers, [----] engagements
"@jfais20 I like $rgnx but one statement is not very accurate age groups that $srpt cant touch: Sarepta plans on filing BLA expansion for [--] yr olds second half of this year which would imo be well ahead of rgnx:"
X Link 2025-02-11T15:59Z [----] followers, [----] engagements
"@edcollins23 @largelylurking @JonU235 @Nicklaus4Kids Good point wonder if he is confusing with Zolgensma which is AAV9- or another GTx they did clinical trials with. $srpt $nvs"
X Link 2025-02-14T12:10Z [----] followers, [---] engagements
"$rhhby $srpt #Elevidys #Brazil https://www.metropoles.com/colunas/grande-angular/crianca-com-doenca-rara-toma-remedio-de-r-17-mi-quase-na-data-limite https://www.metropoles.com/colunas/grande-angular/crianca-com-doenca-rara-toma-remedio-de-r-17-mi-quase-na-data-limite"
X Link 2025-02-14T22:08Z [----] followers, [---] engagements
"@JonU235 $srpt Another key one for EMA : https://www.mdaconference.org/abstract-library/muscle-mri-outcomes-in-patients-with-duchenne-muscular-dystrophy-treated-with-delandistrogene-moxeparvovec-findings-from-embark-part-1/ https://www.mdaconference.org/abstract-library/muscle-mri-outcomes-in-patients-with-duchenne-muscular-dystrophy-treated-with-delandistrogene-moxeparvovec-findings-from-embark-part-1/"
X Link 2025-02-15T05:21Z [----] followers, [---] engagements
"@SteveHa64191820 @Biotech2k1 $srpt (4x) does appear cheap- at least comparing commercial stage companies he listed based on [----] revenue estimates vs market caps: $bbio (30x) $insm (28x) $axsm (10x) $bpmc (6x)"
X Link 2025-02-16T14:14Z [----] followers, [--] engagements
"@SteveHa64191820 @Biotech2k1 $srpt (4x) does appear cheap- at least comparing commercial stage companies he listed based on [----] revenue estimates vs market caps: $bbio (30x) $insm (28x) $axsm (10x) $bpmc (8x)"
X Link 2025-02-16T14:41Z [----] followers, [----] engagements
"Very key point $srpt $rgnx $sldb : @cognivestment @adamfeuerstein @Pharmdca I would be careful to compare it to Elevidys since Elevidys allows viral antibodies up to 1:400 which is pretty high. Solid Regenx and Genethon are all only dosing if no antibodies (like 1:20 or 1:25). Other than that I think Solid is proving to be safe. @cognivestment @adamfeuerstein @Pharmdca I would be careful to compare it to Elevidys since Elevidys allows viral antibodies up to 1:400 which is pretty high. Solid Regenx and Genethon are all only dosing if no antibodies (like 1:20 or 1:25). Other than that I think"
X Link 2025-02-18T22:50Z [----] followers, [----] engagements
"@BiotechAnalysst This is key $srpt first N=4 mean of 81.2% of muscle fibers expressing micro-dystrophin with a mean intensity of 96% at the sarcolemma. Western blot showed a mean expression of 74.3% without fat or fibrosis adjustment and 95.8% with adjustment. And no SAEs. (1/2)"
X Link 2025-02-19T07:43Z [----] followers, [---] engagements
"@BiotechAnalysst (2/2) This similar to $sldb early N=3. And in $srpt larger N trials for Embark/Endeavor %positive fibers stayed similar to sldb but expression was 34% for Embark. So will expression drop for sldb in larger N also Functional data MOST important(see pfe) http://pubmed.ncbi.nlm.nih.gov/32539076/ http://pubmed.ncbi.nlm.nih.gov/32539076/"
X Link 2025-02-19T08:17Z [----] followers, [---] engagements
"@BiotechAnalysst And obviously need larger N to compare safety properly as again srpt reported no SAEs in first N=4. Altho key is whole field has benefited greatly from pooled $srpt $pfe $sldb Genethon safety data/experience: https://www.fiercebiotech.com/biotech/pfizer-sarepta-team-fellow-dmd-gene-therapy-makers-get-bottom-adverse-events https://www.fiercebiotech.com/biotech/pfizer-sarepta-team-fellow-dmd-gene-therapy-makers-get-bottom-adverse-events"
X Link 2025-02-19T08:19Z [----] followers, [---] engagements
"@Sports_bios (Contd) Its obvious all [--] of these companies pre selected for most ideal patients to raise critical $ at time of initial N=3/4 data. Will come down to much larger trials (N=50 to 100) and will come down to functional data. Jmo"
X Link 2025-02-19T17:57Z [----] followers, [---] engagements
"$rhhby $srpt :"
X Link 2025-02-20T03:08Z [----] followers, [--] engagements
"Wonder if its pfe thats paying stat/AF to dump all over GTx since pfe completely struck out in GTx commercially in embarrassing fashion $nvs $srpt $bmrn $crsp etc: $PFE stops commercialization of hemophilia gene therapy Beqvez that has been approved in April [----] Beqvez is a one-time therapy that was approved in the U.S. last year for the treatment of adults with moderate to severe hemophilia B. $RHHBY $SGMO Beqvez was priced at $3.5 $PFE stops commercialization of hemophilia gene therapy Beqvez that has been approved in April [----] Beqvez is a one-time therapy that was approved in the U.S."
X Link 2025-02-21T14:00Z [----] followers, [----] engagements
"@occams_tooth @PersimmonTI $nvs and Zolgensma just fine too"
X Link 2025-02-21T20:46Z [----] followers, [--] engagements
"@jfais20 $srpt got [--] CNS programs (HD and [--] ataxias) from $arwr in their existing partnership. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-global-licensing-and https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-global-licensing-and"
X Link 2025-06-06T15:17Z [----] followers, [---] engagements
"@jfais20 (Contd) Additionally Sarepta and Arrowhead have entered into a discovery collaboration for up to six additional muscle cardiac and/or CNS targets using Arrowheads novel delivery technologies. $srpt $arwr $rna $dyn $alny"
X Link 2025-06-06T15:19Z [----] followers, [---] engagements
"@LTbioinvestor this data is similar to Elevidys data imo. $rna has bigger market cap than $srpt ; no revenue. And $arwr / $srpt have lower siRNA dose in FSHD"
X Link 2025-06-09T14:20Z [----] followers, [----] engagements
"If reasonable now ask yourself what % of the $arwr pipeline / platform does $srpt own (keeping in mind $rna market cap.) $ARWR is a great example of why public platforms arent getting acquired. They cost too much Capstan set a benchmark with its $2.1B buyout ($500M pre-acqu post money valuation per PB) a 4x step-up on private valuation. Now compare that to $ARWR: 🔹Current market cap: $2B $ARWR is a great example of why public platforms arent getting acquired. They cost too much Capstan set a benchmark with its $2.1B buyout ($500M pre-acqu post money valuation per PB) a 4x step-up on private"
X Link 2025-07-01T23:55Z [----] followers, [---] engagements
"@BowTiedBiotech IMO $rna market cap (first in class muscle targets but likely not best in class) completely backs up this above valuation estimate $arwr $srpt $ARWR (L) TRiM muscle programs use 1/10th - 1/50th the amount of total drug vs $DYN and $RNA Could be benefits from safety standpoint (also potentially allows $SRPT to toggle to quarterly subQ administration vs every 6-8 week IV) Good pace of enrollment data perhaps 2H [--] 🧐 https://t.co/Ava6uXsi6u $ARWR (L) TRiM muscle programs use 1/10th - 1/50th the amount of total drug vs $DYN and $RNA Could be benefits from safety standpoint (also"
X Link 2025-07-02T00:37Z [----] followers, [---] engagements
"Is this one reason why $arwr up big today $200M coming soon from $srpt They expanded their DM1 P1/2 trial to a bigger enrollment than what $rna had in P1/2 and I believe added functional outcomes that rna did not have in P1/2 https://clinicaltrials.gov/study/NCT06138743cond=DM1&term=Arrowhead&rank=1&tab=history&a=9&b=10#version-content-panel https://clinicaltrials.gov/study/NCT06138743cond=DM1&term=Arrowhead&rank=1&tab=history&a=9&b=10#version-content-panel"
X Link 2025-08-13T16:45Z [----] followers, [---] engagements
"(Contd) $rna I believe had DLT (4mg) in their P1/2. So something to compare for $arwr / $srpt in DM1"
X Link 2025-08-13T16:49Z [----] followers, [---] engagements
"@RNAiAnalyst How so Because they accepted $50M in their own stock as payment Or do you think arwr was part of $174M block transaction"
X Link 2025-08-14T04:50Z [----] followers, [---] engagements
"@Amitpat42375370 @blippd @semodough $cytk https://www.royaltypharma.com/news/royalty-pharma-and-cytokinetics-announce-expanded-strategic-funding-collaboration-totaling-up-to-575-million-to-support-commercial-launch-of-aficamten-and-to-advance-rd-pipeline/ https://www.royaltypharma.com/news/royalty-pharma-and-cytokinetics-announce-expanded-strategic-funding-collaboration-totaling-up-to-575-million-to-support-commercial-launch-of-aficamten-and-to-advance-rd-pipeline/"
X Link 2025-08-30T15:45Z [----] followers, [---] engagements
"@Sanctuary_Bio @MauriceOnTW They have early safety signals. Will likely get worse in P3. $sldb $rgnx $srpt"
X Link 2025-09-03T17:53Z [----] followers, [---] engagements
"@yachmod @edcollins23 @MauriceOnTW @Sanctuary_Bio @BiotechObserver May very well still but $srpt took it off their pipeline page. Maybe dont want to step on Roches toes Or they terminated deal Not clear to me. But my guess is gnt0004 has real value as Genethon may be ahead of both $rgnx $sldb. https://www.genethon.com/genethon-to-launch-pivotal-trial-in-europe-of-gnt0004-a-low-dose-microdystrophin-gene-therapy-for-duchenne-muscular-dystrophy/"
X Link 2025-09-03T22:16Z [----] followers, [---] engagements
"@Sanctuary_Bio @BiotechObserver I dont think thats true. Again back to CEOs disclosure problems and keeping in mind the FDA knew about that one death month before all the FDA srpt drama"
X Link 2025-09-04T17:39Z [----] followers, [---] engagements
"@TTRAmyloid But yet $rna insiders dumped $20M the last month. And data is far from slam dunk for stat sig in P3: $arwr $dyn $pepg https://www.aviditybiosciences.com/sites/default/files/2025-04/Final-NMSG-2024-FORTITUDE-Poster-16SEP24.pdf https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf https://www.aviditybiosciences.com/sites/default/files/2025-04/Final-NMSG-2024-FORTITUDE-Poster-16SEP24.pdf"
X Link 2025-09-04T23:24Z [----] followers, [---] engagements
"@TTRAmyloid And $rna DLT (clinical hold) in DM1 is definitely something to watch for vs competitors. https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf"
X Link 2025-09-04T23:31Z [----] followers, [---] engagements
"@TTRAmyloid Has $rna released any functional data on its exon [--] skipper to show that it works (1/2) https://www.prnewswire.com/news-releases/avidity-biosciences-announces-positive-topline-del-zota-data-demonstrating-consistent-statistically-significant-improvements-in-dystrophin-exon-skipping-and-creatine-kinase-in-people-living-with-duchenne-muscular-dystrophy-amena-302402470.html"
X Link 2025-09-05T06:02Z [----] followers, [---] engagements
"@TTRAmyloid (2/2) $rna $srpt $dyn $wve $pepg #NS Exon skip tough from a regulatory standpt in terms of grind it out piecemeal approvals; many skippers have small TAMs (ie exon 44); v crowded in terms of competition. Good summary on DMD exon skipping field/competition: https://musculardystrophynews.com/exon-skipping-for-duchenne-muscular-dystrophy/utm_source=MD&utm_campaign=1f43b91c7c-Email_ENL_US_MD&utm_medium=email&utm_term=0_ea166dad7b-1f43b91c7c-72135529"
X Link 2025-09-05T06:19Z [----] followers, [---] engagements
"@TTRAmyloid To look at actual data $rna Del Zota data seems pretty similar to $srpt Elevidys vs natural history In fact this data favors Elevidys (1/2 contd): Also: https://www.cgtlive.com/view/sarepta-dmd-gene-therapy-elevidys-sustained-benefit-updated-phase-3-data https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-presents-data-american-society-gene-cell https://www.cgtlive.com/view/sarepta-dmd-gene-therapy-elevidys-sustained-benefit-updated-phase-3-data"
X Link 2025-09-10T13:59Z [----] followers, [----] engagements
"@TimCarmichael3 $rna now has discontinuations in [--] of [--] key early phase programs. Not necessarily a platform safety issue but something to keep an eye on.: DMD DM1 https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf https://www.prnewswire.com/news-releases/avidity-biosciences-del-zota-demonstrated-reversal-of-disease-progression-across-key-functional-endpoints-in-explore44-and-explore44-ole-phase-12-trial-in-people-living-with-dmd44-302552339.html"
X Link 2025-09-10T21:28Z [----] followers, [---] engagements
"@scamsweeper (2/2) I believe $arwr has gotten to [--] and [--] mg/kg and $pepg to at least [--] mg/kg . Tiny Pepgen claims better DM1 splicing data than $rna on single dose"
X Link 2025-09-12T16:39Z [----] followers, [----] engagements
"@scamsweeper Compare pepgen splicing data to avidity about same imo: https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA-DMPK-Spliceopathy-Poster-30x42in_V4.0_Final-for-Upload.pdf https://finance.yahoo.com/news/pepgen-announces-positive-initial-results-120000624.html https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA-DMPK-Spliceopathy-Poster-30x42in_V4.0_Final-for-Upload.pdf https://finance.yahoo.com/news/pepgen-announces-positive-initial-results-120000624.html"
X Link 2025-09-12T17:00Z [----] followers, [---] engagements
"This is true with one time GTxs. But key is an overall fatality rate of 1% for $srpt; a one time tx. Juxtapose this vs chronic (lifetime) RNA txs that are seeing discontinuations (and related clinical holds) for $rna $dyn $pepg etc in multiple muscular dystrophy dxs"
X Link 2025-09-22T14:33Z [----] followers, [----] engagements
"@cal89511029 $srpt indicated they would file BLA for Srp-9003 before year end: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-strategic-restructuring-and https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-strategic-restructuring-and"
X Link 2025-09-23T17:42Z [----] followers, [---] engagements
"@blippd @scamsweeper (2/2) so safety is in question for their platform and all comes down to MAD. But imo $pepg has a chance as $rna most advanced in DM1 but efficacy+safety pretty mediocre. Also $arwr DM1 data imminent and also has chance at BIC as think theyve gotten well beyond 4mg/kg"
X Link 2025-09-24T21:08Z [----] followers, [----] engagements
"@blippd @scamsweeper Also only seen splicing data from $pepg If so really need to see DM1 functional data in MAD $rna $dyn $arwr"
X Link 2025-09-24T21:11Z [----] followers, [---] engagements
"@semodough I think $srpt only saw hypoMG in their MAD study for Srp-5051. So the sad study doesnt mean much no $pepg already seen [--] clinical holds. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-update-srp-5051-treatment-duchenne https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-update-srp-5051-treatment-duchenne"
X Link 2025-09-24T22:08Z [----] followers, [---] engagements
"@ShafferBiotech And why no functional data similar to Avidity (which imo was pretty mediocre for efficacy and safety) if splicing data so superior https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf"
X Link 2025-09-24T23:30Z [----] followers, [---] engagements
"@ShafferBiotech $pepg $rna $dyn : $PEPG the functional data at day [--] doesnt seem too different from the $RNA data. Interestingly the functional data only shown upto day [--] for lower [--] doses though based on the study design they should have week [--] biopsy data + other longer term data by now for those doses https://t.co/zc6CFSZQdL $PEPG the functional data at day [--] doesnt seem too different from the $RNA data. Interestingly the functional data only shown upto day [--] for lower [--] doses though based on the study design they should have week [--] biopsy data + other longer term data by now for"
X Link 2025-09-25T10:22Z [----] followers, [---] engagements
"@RNAiAnalyst @JamesMclaw70633 (Contd) see this thread ( $qure $ions $arwr $srpt etc): @Hall8Jack @RNAiAnalyst @JamesMclaw70633 My understanding is that you want to avoid knocking down wild type HTT. Ideally want mutant specific knock-down. As I understand it this was one of the lessons from the failure of Roche / Ionis Tominersen @Hall8Jack @RNAiAnalyst @JamesMclaw70633 My understanding is that you want to avoid knocking down wild type HTT. Ideally want mutant specific knock-down. As I understand it this was one of the lessons from the failure of Roche / Ionis Tominersen"
X Link 2025-09-26T10:04Z [----] followers, [----] engagements
"@RNAiAnalyst Will be tough to compare as the ASOs and siRNA approaches will be running PBO controlled trials and no brain surgery- as opposed to $qure. $ions $alny $arwr $srpt"
X Link 2025-09-26T13:29Z [----] followers, [---] engagements
"@RNAiAnalyst (Contd) so I am sincerely hoping that many HD patients have the legit choice of a COMBO therapy of both gene tx and RNA tx $qure $ions $alny $arwr $srpt etc"
X Link 2025-09-26T13:37Z [----] followers, [---] engagements
"$tsha $ngne #Rett #genetherapy $xbi"
X Link 2025-10-02T16:51Z [----] followers, [---] engagements
"@newerraa And I would guess $arwr / $srpt (Srp-1003) DM1 trial will be updated also in the next several days"
X Link 2025-10-07T16:42Z [----] followers, [---] engagements
"@newerraa Do you have any expectations from the ARO-MMP7 IPF trial $arwr has proven they can get lung exposure with ARO-RAGE but $srpt doesnt mention ARO-MMP7 anymore"
X Link 2025-10-07T16:47Z [----] followers, [----] engagements
"@planert41 IMO eye is on wrong ball. This data matters of course but siRNA data (FSHD/DM1) matters more.again imo $srpt $arwr"
X Link 2025-10-07T22:45Z [----] followers, [---] engagements
"@RNAiAnalyst Did you read this AMT-130 apparently lowers both WT and mutant HTT. Very similar to $arwr / $srpt approach. Obviously $qure intrinsically brain targeted but arwr likely has brain targeting advantages (subQ vs IT) over $ions $alny $wve etc. Should be interesting. I've finally digested the data that Uniqure presented on AMT-130 their gene therapy for Huntington's disease which just announced a positive trial readout with 75% slowing of disease progression. Here is a blog post with my take on the announcement: https://t.co/7Rz1hGQ0X2 I've finally digested the data that Uniqure"
X Link 2025-10-08T20:27Z [----] followers, [----] engagements
"@RNAiAnalyst $qure $arwr :"
X Link 2025-10-08T20:45Z [----] followers, [---] engagements
"@yachmod Think you mean Genethon $srpt used to own US rights to this. Not sure where that stands now: https://www.cgtlive.com/view/genethon-gene-therapy-gnt0004-continues-show-potential-dmd https://www.cgtlive.com/view/genethon-gene-therapy-gnt0004-continues-show-potential-dmd"
X Link 2025-10-11T01:01Z [----] followers, [---] engagements
"@BiotechAnalysst So thats [--] Imlifidase studies from $hnsa - one with Genethon and this from $srpt : Hansa x $SRPT progress. https://t.co/3efOy4Tpoq Hansa x $SRPT progress. https://t.co/3efOy4Tpoq"
X Link 2025-10-14T10:21Z [----] followers, [----] engagements
"$arwr $srpt siRNA IPF trial completed: https://clinicaltrials.gov/study/NCT05537025cond=IPF&term=Arrowhead&rank=1&tab=history&a=23&b=24#version-content-panel https://clinicaltrials.gov/study/NCT05537025cond=IPF&term=Arrowhead&rank=1&tab=history&a=23&b=24#version-content-panel"
X Link 2025-10-15T22:21Z [----] followers, [----] engagements
"@newerraa Interesting $srpt has terminated or withdrawn several trials recently due to business decision (recent restructuring) in LGMDs and Elevidys/plasmapheresis but has not made similar decision in IPF . $arwr "
X Link 2025-10-16T03:50Z [----] followers, [----] engagements
"$xbi $ibb : FDA Commissioner Makary speaking at White House event announces that [--] companies are receiving his Priority Vouchers. More details coming soon but EMD Serono has snagged one. FDA Commissioner Makary speaking at White House event announces that [--] companies are receiving his Priority Vouchers. More details coming soon but EMD Serono has snagged one"
X Link 2025-10-16T20:49Z [----] followers, [---] engagements
"$xbi $ibb : https://www.fda.gov/news-events/press-announcements/fda-awards-first-ever-national-priority-vouchers-nine-sponsors https://www.fda.gov/news-events/press-announcements/fda-awards-first-ever-national-priority-vouchers-nine-sponsors"
X Link 2025-10-16T21:19Z [----] followers, [---] engagements
"@cal89511029 Would this be for LGMD2e gene therapy as has been conjectured $srpt will file bla soon"
X Link 2025-10-20T13:44Z [----] followers, [---] engagements
"@rezfszubagoly @cal89511029 So this might be for a siRNA product (DM1 FSHD IPF) $srpt $arwr"
X Link 2025-10-20T14:27Z [----] followers, [---] engagements
"@Bionerd51 @BruceHarri19541 You claim this based on what precisely $srpt $arwr #ipf"
X Link 2025-10-21T16:41Z [----] followers, [---] engagements
"@suspendedJoey They were supposed to file the HD ARO-HTT IND before the end of the year. Have you seen anything $srpt $arwr"
X Link 2025-10-23T12:26Z [----] followers, [---] engagements
"@Biobeats99 $rhhby $104M this Q (64% growth yoy) 2nd highest since Elevidys exUS launch ( $srpt ) :"
X Link 2025-10-23T19:46Z [----] followers, [----] engagements
"@cal89511029 The 221M chf is for the entire year to date or for [--] Qs. This Q was [---] million chf"
X Link 2025-10-24T02:43Z [----] followers, [---] engagements
"@newerraa The 221M chf is for the entire year to date or for [--] Qs. This Q was [---] million chf"
X Link 2025-10-24T02:43Z [----] followers, [---] engagements
"@RNAiAnalyst @newerraa @Roche @Stocktwits There were shipment stops in Japan (there was a slide on regional Roche sales where it appeared Elevidys had no Japan sales in Q3) Brazil and I think a couple Middle Eastern countries. If so not sure when resume(d). I think current US Elevidys consensus estimate stands at $100M"
X Link 2025-10-24T10:20Z [----] followers, [---] engagements
"@edcollins23 @RNAiAnalyst @newerraa @Roche @Stocktwits Cant argue with you but Roche got to $130M this quarter and Elevidys was/is paused in several of the 9/10 exUS countries where Elevidys was approved. I think US sales have always been greater than exUS. For example- last Q US=$280M; exUS=$90M. $srpt $rhhby"
X Link 2025-10-24T14:10Z [----] followers, [---] engagements
"@PersimmonTI @MichelleF_Davis Why would $rna sell out for like $10B If they get both the DM1 and FSHD markets (ea TAM $1-2B) that should be worth $15-20B market cap. Look at $alny mc of $60B"
X Link 2025-10-26T15:13Z [----] followers, [---] engagements
"Interesting $nvs paid $9B for Zolgensma (Avexis) which does about $1B per year. Now $10B for $rna "
X Link 2025-10-26T15:47Z [----] followers, [---] engagements
"And $rna results are not necessarily unprecedented in any of the indications. $pepg / $dyn showing similar splicing (and functional improvements ) in DM1 and/or FSHD; rna showing similar fold increase (key to compare to baseline) in minidystrophin as other exon skippers for DMD"
X Link 2025-10-26T15:55Z [----] followers, [---] engagements
"@RNAiAnalyst @Gabrielmendesnu Why is $rna DM1 sure thing over FSHD Didnt DM1 have a discontinuation in small early trial Who knows how that manifests itself in much larger P3. $rna $dyn $pepg etc"
X Link 2025-10-26T16:35Z [----] followers, [---] engagements
"@RNAiAnalyst They might be closer for data in FSHD $RNA $dyn $pepg $arwr SRP [----] Update $SRPT https://t.co/sK47i8vak5 SRP [----] Update $SRPT https://t.co/sK47i8vak5"
X Link 2025-10-26T18:27Z [----] followers, [----] engagements
"@commonsenseplay Judging by $nvs / $rna deal imo $srpt s DM1 FSHD [--] Ataxias Huntingtons and IPF $arwr siRNA programs have far far more value than Elevidys "
X Link 2025-10-26T18:50Z [----] followers, [----] engagements
"@PikaCapital (1/3 $rna $dyn $arwr $srpt ) Its a very interesting question if you read this: $ARWR (L) TRiM muscle programs use 1/10th - 1/50th the amount of total drug vs $DYN and $RNA Could be benefits from safety standpoint (also potentially allows $SRPT to toggle to quarterly subQ administration vs every 6-8 week IV) Good pace of enrollment data perhaps 2H [--] 🧐 https://t.co/Ava6uXsi6u $ARWR (L) TRiM muscle programs use 1/10th - 1/50th the amount of total drug vs $DYN and $RNA Could be benefits from safety standpoint (also potentially allows $SRPT to toggle to quarterly subQ administration"
X Link 2025-10-26T21:54Z [----] followers, [---] engagements
"@PikaCapital (2/3 $rna $dyn $arwr $srpt ) or listen to this presentation from RBC: https://kvgo.com/rbc/arrowhead-pharmaceuticals-inc-may-2025 https://kvgo.com/rbc/arrowhead-pharmaceuticals-inc-may-2025"
X Link 2025-10-26T21:55Z [----] followers, [---] engagements
"@PikaCapital (3/3 $rna $dyn $arwr $srpt ) or read this: https://ir.arrowheadpharma.com/static-files/c8100bbd-963d-4275-9233-c0b3f18f6d31 https://ir.arrowheadpharma.com/static-files/c8100bbd-963d-4275-9233-c0b3f18f6d31"
X Link 2025-10-26T21:57Z [----] followers, [---] engagements
"@PikaCapital Key claims via slides $rna $dyn vs $arwr :"
X Link 2025-10-26T22:03Z [----] followers, [----] engagements
"@sonichedgefund Mildly interesting - $nvs PR wording : The Avidity programs feature potential first-in-class late-stage disease-modifying therapies. They dont word it potentially best in class. $rna $dyn $arwr etc"
X Link 2025-10-26T23:49Z [----] followers, [---] engagements
"@DickMedChem Typical $wve - early data hype; not enough meat on the bone"
X Link 2025-10-29T20:39Z [----] followers, [---] engagements
"@financebully @avidresearch Do you think $wve will even be able to raise $ off of this limited data reveal"
X Link 2025-10-29T23:25Z [----] followers, [---] engagements
"@Longroaderstar Looks like $arwr tacking on more market cap gains off of $wve INHBE obesity update than wave is. #ALK7"
X Link 2025-10-30T15:11Z [----] followers, [---] engagements
"@DeuceVault $srpt :"
X Link 2025-10-30T20:22Z [----] followers, [----] engagements
"@BiomedicalRX @PDUFA_Pulse lol $arwr all about INHBE / ALK7 now"
X Link 2025-10-31T23:41Z [----] followers, [---] engagements
"$srpt $arwr : $ARWR adds a couple of arms to its P1/2a myotonic dystrophy study pushes out primary completion date [--] months https://t.co/fM1G9GFRpG $ARWR adds a couple of arms to its P1/2a myotonic dystrophy study pushes out primary completion date [--] months https://t.co/fM1G9GFRpG"
X Link 2025-11-11T19:08Z [----] followers, [----] engagements
"Looks like $arwr $srpt got to subQ for DM1. Discussed at RBC presentation here at [--] min mark: if we can toggle to subQ from IV quarterly then could be transformational vs avidity/dyne. And here: https://kvgo.com/rbc/arrowhead-pharmaceuticals-inc-may-2025 $ARWR (L) TRiM muscle programs use 1/10th - 1/50th the amount of total drug vs $DYN and $RNA Could be benefits from safety standpoint (also potentially allows $SRPT to toggle to quarterly subQ administration vs every 6-8 week IV) Good pace of enrollment data perhaps 2H [--] 🧐 https://t.co/Ava6uXsi6u"
X Link 2025-11-11T19:24Z [----] followers, [----] engagements
"Corrected: at about the [--] min mark; not the [--] min mark for the RBC presentation $arwr $srpt"
X Link 2025-11-11T19:33Z [----] followers, [---] engagements
"Just a reminder avidity P3 DM1 trial IV. . Apparently $arwr $srpt just got to subQ for their P1/2 trial. Could be a very significant differentiator. http://clinicaltrials.gov/study/NCT06411 http://clinicaltrials.gov/study/NCT06411"
X Link 2025-11-11T20:29Z [----] followers, [---] engagements
"@DM1research Avidity/ $nvs hit dose limiting tox at [--] mg/kg I believe for both DM1 and FSHD trials. Whereas $arwr $srpt I think has reached [--] mg/kg cohorts for both DM1/FSHD. And hopefully subQ vs IV"
X Link 2025-11-11T21:31Z [----] followers, [---] engagements
"@veevi24 @DM1research Unfortunately the corresponding Arrowhead trial for FSHD is still only IV arms. But since Arrowhead DM1 trial just added subQ arms it is very possible FSHD will do also since they are similar drugs. https://clinicaltrials.gov/study/NCT06131983cond=FSHD&term=Arrowhead&rank=1 https://clinicaltrials.gov/study/NCT06131983cond=FSHD&term=Arrowhead&rank=1"
X Link 2025-11-12T03:54Z [----] followers, [--] engagements
"@investvee Hoping this is good news for $rhhby $srpt Envol trial and potential approval for boys under [--] who should benefit even more than 4+ ambulatory : https://clinicaltrials.gov/study/NCT06128564cond=DMD&term=Envol&rank=1 https://clinicaltrials.gov/study/NCT06128564cond=DMD&term=Envol&rank=1"
X Link 2025-11-14T19:00Z [----] followers, [---] engagements
"@investvee (Contd) altho have no idea how it affects $srpt BLA submission for SRP-9003 for LGMD2e"
X Link 2025-11-14T19:04Z [----] followers, [---] engagements
"@RNAiAnalyst $wve doesnt even have a DM1 program. $rna $dyn $arwr $pepg $vrtx"
X Link 2025-11-15T19:04Z [----] followers, [---] engagements
"@RNAiAnalyst @JamesMclaw70633 And in terms of potency claim you should listen to $arwr RBC presentation at [--] min mark. Claims very potent and 1/10th to 1/50th of total drug vs $rna $dyn . We shall see https://kvgo.com/rbc/arrowhead-pharmaceuticals-inc-may-2025 https://kvgo.com/rbc/arrowhead-pharmaceuticals-inc-may-2025"
X Link 2025-11-15T19:23Z [----] followers, [----] engagements
"@RNAiAnalyst @JamesMclaw70633 IMO MAD Functional data and safety much more important than SAD splicing data. Hence $pepg only $300m mc $rna $dyn $arwr"
X Link 2025-11-15T19:48Z [----] followers, [---] engagements
"@RNAiAnalyst @JamesMclaw70633 Abs typically 15-50x larger than small protein; FAbs less so. So not that complicated to adjust for relative potency"
X Link 2025-11-15T19:57Z [----] followers, [--] engagements
"@RNAiAnalyst @JamesMclaw70633 Be careful judging from their DMD programs $pepg may have a platform delivery tox issue/disadvantage (kidney/hypoMg) vs $rna . Which is particularly concerning as avidity had a clinical hold in DM1"
X Link 2025-11-15T21:04Z [----] followers, [---] engagements
"@BiotechAnalysst Pull an additional [---] pts from commercial TAM Here says it is an observational study: FDA also required $srpt to run a post-marketing observational study with [---] patients subjected to periodic liver function tests for [--] months after Elevidys tx https://www.biospace.com/fda/fda-imposes-boxed-warning-narrows-patient-pool-for-sareptas-duchenne-gene-therapy-elevidys https://www.biospace.com/fda/fda-imposes-boxed-warning-narrows-patient-pool-for-sareptas-duchenne-gene-therapy-elevidys"
X Link 2025-11-17T14:54Z [----] followers, [----] engagements
"@BiotechAnalysst (Contd) Which i think is Expedition or Endure which is either from commercial pts or pts during a previous clinical study received Elevidys. $srpt https://clinicaltrials.gov/study/NCT05967351cond=DMD&term=Expedition&rank=1 https://clinicaltrials.gov/study/NCT06270719cond=DMD&term=Endure&rank=1 https://clinicaltrials.gov/study/NCT05967351cond=DMD&term=Expedition&rank=1 https://clinicaltrials.gov/study/NCT06270719cond=DMD&term=Endure&rank=1"
X Link 2025-11-17T15:04Z [----] followers, [---] engagements
"@RNAiAnalyst But yet you are betting on $pepg and $dyn over $arwr in DM1. Should be very interesting"
X Link 2025-11-18T21:02Z [----] followers, [---] engagements
"@BikeRieder At [--] min mark - $arwr in FSHD and DM1 we think are great drugs trials progressing well and DM1 should trigger $200M payment from $srpt by end of 2025"
X Link 2025-11-20T04:01Z [----] followers, [---] engagements
"@TrendSpider The day trading trump boys couldnt dump their crypto holdings fast enough lol"
X Link 2025-11-22T19:22Z [----] followers, [---] engagements
"@ArtsStocks If true how did $rna (BO for $12B by $nvs ) $dyn $pepg $vrtx expand into DM1 and/or FSHD w siRNAs and how did $wve expand into INHBE/obesity using siRNAs They have obtained no IP from $ions $alny . All of these companies ( $arwr ) have their own IP based on conjugated delivery"
X Link 2025-11-24T16:49Z [----] followers, [---] engagements
"@ArtsStocks And yes $arwr when they obtained $nvs and $rhhby RNAi assets over [--] years ago had transferred $alny licensing deals which gave arwr freedom to operate" in the RNAi field"
X Link 2025-11-24T16:52Z [----] followers, [---] engagements
"@ArtsStocks So are you saying $nvs wasted their time and $ with $rna BO"
X Link 2025-11-24T17:04Z [----] followers, [---] engagements
"@ArtsStocks Uh huh $nvs blew their DD. IMO you are wildly over interpreting the plozasiran litigation. Which is likely tenuous in and of itself $ions $arwr"
X Link 2025-11-24T17:21Z [----] followers, [---] engagements
"@ArtsStocks You realize the conjugated delivery that $arwr uses for plozasiran is different from the conjugated delivery they use for lung targets; CNS targets and muscle targets; etc. etc. Just curious are you trying to argue $ions has broad claims for delivery to all those targets"
X Link 2025-11-24T17:43Z [----] followers, [---] engagements
"@BioTecIsFuture There are no effective drugs for DM1. Why do you think $nvs BO $rna for $12B Best in class will likely peak north of $2B per year- note these #s based on 10% market share:"
X Link 2025-11-24T17:50Z [----] followers, [---] engagements
"@commonsenseplay Also worth noting avidity ( $nvs BO $12B) hit dose limiting tox at [--] mg/kg in their P1/2 trial. $arwr $srpt"
X Link 2025-11-24T20:07Z [----] followers, [---] engagements
"@commonsenseplay Avidity basically stopped at 4mg/kg. $arwr $srpt now at [--] and [--] mg/kg. https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf https://www.aviditybiosciences.com/sites/default/files/2025-04/WMS-2023_MARINA_Data_Encore_Poster_30x42in_v6.0_Final-for-Upload.pdf"
X Link 2025-11-24T20:09Z [----] followers, [---] engagements
"@commonsenseplay Also $arwr $srpt added subQ cohort to IV cohorts; avidity only IV: https://clinicaltrials.gov/study/NCT06138743cond=DM1&term=Arrowhead&rank=1 https://clinicaltrials.gov/study/NCT06138743cond=DM1&term=Arrowhead&rank=1"
X Link 2025-11-24T20:13Z [----] followers, [---] engagements
"@EasterWing2230 @BiotechAnalysst @yachmod How did the [--] and [--] bonds do today after announcing $200M arwr payment I cant see accurate quotes"
X Link 2025-11-24T21:48Z [----] followers, [--] engagements
"From Dr. Jerry Mendell at NCH #Itvisma / #Zolgensma $nvs . He was also the inventor of $srpt #Elevidys:"
X Link 2025-11-24T23:44Z [----] followers, [---] engagements
"@BowTiedBiotech lol no mention of the debt (ie Celgene) $bmy"
X Link 2025-11-24T23:56Z [----] followers, [---] engagements
"@aakashg0 Interesting theory at same time $nvda / $crwv / OpenAI etc. are deflating we have to now pump $goog AI"
X Link 2025-11-25T00:35Z [----] followers, [---] engagements
"@AIStockSavvy This is worded very poorly. $srpt already said that cohort [--] at 6MPK has started dosing so logically cohort [--] is next at [--] MPK: They also dont mention that $arwr just added new subcutaneous arms in addition to the previous IV arms. Very important detail: https://finance.yahoo.com/news/sarepta-provides-progress-srp-1003-123000296.html https://finance.yahoo.com/news/sarepta-provides-progress-srp-1003-123000296.html"
X Link 2025-11-25T14:43Z [----] followers, [---] engagements
"@AIStockSavvy (Contd) . Here was the original design. So they added a 4.5mpk cohort and then added a subQ arm: . So yes $arwr $srpt made a couple key changes to original design https://ir.arrowheadpharma.com/static-files/c8100bbd-963d-4275-9233-c0b3f18f6d31 https://clinicaltrials.gov/study/NCT06138743cond=DM1&term=Arrowhead&rank=1 https://ir.arrowheadpharma.com/static-files/c8100bbd-963d-4275-9233-c0b3f18f6d31 https://clinicaltrials.gov/study/NCT06138743cond=DM1&term=Arrowhead&rank=1"
X Link 2025-11-25T14:52Z [----] followers, [---] engagements
"@TimCarmichael3 This is a very good point- at this stage of trials $rna (DM1) $dyn (DMD) and $pepg (DMD) all had clinical holds with similar drugs. Not $arwr $srpt in DM1 and FSHD SO FAR"
X Link 2025-11-25T16:44Z [----] followers, [---] engagements
"@TheProfessor_85 $arwr has grown to my largest position but when you look at economics of $srpt deal - if DM1 and/or FSHD hit (ie best in class) - srpt will be worth more than arwr just mo"
X Link 2025-11-25T21:40Z [----] followers, [---] engagements
"@doepke_michel So besides $mdgl - also add $arwr to this potential BO list Or is arwr simply the new spec obesity ticker 🤷🏽"
X Link 2025-11-26T15:25Z [----] followers, [---] engagements
"@ArtsStocks Market certainly doesnt seem as worried about the $ions $arwr litigation risk as you are ;-)"
X Link 2025-11-26T15:45Z [----] followers, [---] engagements
"@BiotechAnalysst @SangamoTx So [--] other interested parties in $rna beside $nvs . Any chance any of them moved onto $arwr or $dyn $srpt etc $XBI how a buyout deal is negotiated: here: $NVS for $RNA RNA contacts [--] third parties [--] of them interested. FYI CC @sangamotx https://t.co/uhXjlvaBsl $XBI how a buyout deal is negotiated: here: $NVS for $RNA RNA contacts [--] third parties [--] of them interested. FYI CC @sangamotx https://t.co/uhXjlvaBsl"
X Link 2025-11-26T16:33Z [----] followers, [---] engagements
"@titteridiot1 @Singlemalt_18 @MorganStanley Very interesting: . Theyve already partnered aro-snca (PD) w $nvs and aro-HTT (HD) w $srpt for CNS. Aro-mapt next In this presentation they seemed most excited about AD: https://ir.arrowheadpharma.com/static-files/c24adf87-2bec-4068-9d55-38da5058c5c9 https://clinicaltrials.gov/study/NCT07221344term=Aro-mapt&rank=1&tab=history https://ir.arrowheadpharma.com/static-files/c24adf87-2bec-4068-9d55-38da5058c5c9 https://clinicaltrials.gov/study/NCT07221344term=Aro-mapt&rank=1&tab=history"
X Link 2025-11-26T17:03Z [----] followers, [---] engagements
"@titteridiot1 @Singlemalt_18 @MorganStanley And looks like $vktx has been dropped for $arwr for obesity spec. Make any sense $wve"
X Link 2025-11-26T17:06Z [----] followers, [---] engagements
"Its all piling up fairly positive for $arwr . FDA validated plozasiran/TRIM. Obesity data soon so $vktx etc spec obesity $ flowing toward arwr. Signed big subQ CNS/PD deal with $nvs . Also have subQ CNS assets in Ataxia; HD ( $srpt ) and AD (unpartnered so additional upfront $ ). Just got $200M payment from srpt for DM1 (and FSHD). Dimer POC Lung target progress Etc etc"
X Link 2025-11-26T20:50Z [----] followers, [----] engagements
"@avidresearch @JacobPlieth $srpt v $capr - apples to oranges but :"
X Link 2025-12-03T17:50Z [----] followers, [---] engagements
"$arwr +12% today presented at PS Conference; worth a listen (also $srpt $nvs ). Was an RNA panel discussion afterward: https://event.webcasts.com/viewer/event.jspei=1742003&tp_key=742b79bc3d https://event.webcasts.com/viewer/event.jspei=1742003&tp_key=742b79bc3d"
X Link 2025-12-03T21:00Z [----] followers, [----] engagements
"$srpt / Endeavor cohort 8/sirolimus related (also $rgnx $sldb etc #GeneTherapy ) : https://musculardystrophynews.com/news/3-drug-cocktail-boosts-dmd-gene-therapy-effectiveness-mice/mc_euid=9b25abc317&utm_source=MD&utm_campaign=a6a5100400-Email_ENL_US_MD&utm_medium=email&utm_term=0_ea166dad7b-a6a5100400-72135529 https://musculardystrophynews.com/news/3-drug-cocktail-boosts-dmd-gene-therapy-effectiveness-mice/mc_euid=9b25abc317&utm_source=MD&utm_campaign=a6a5100400-Email_ENL_US_MD&utm_medium=email&utm_term=0_ea166dad7b-a6a5100400-72135529"
X Link 2025-12-04T21:16Z [----] followers, [---] engagements
"@BikeRieder Little bit surprised $nvs went with $arwr deal for aro-snca/PD instead of aro-mapt/AD. Do you think aro-mapt too expensive asset or nvs saw higher prob of success for aro-snca $Srpt also passed on aro-mapt (in favor of Ataxia1-3; HD CNS assets) so same type of question"
X Link 2025-12-07T13:53Z [----] followers, [---] engagements
"@LSinnv @BikeRieder CNS SC Programs: aro-mapt/AD (arwr) aro-atxn1/Ataxia1 aro-atxn3/Ataxia3 aro-htt/HD (srpt) aro-snca/PD (nvs)"
X Link 2025-12-07T19:08Z [----] followers, [---] engagements
"@richtrades100 @biopharmacaster $dyn safety for x51 looks worse than $srpt . Will be key consideration"
X Link 2025-12-08T11:53Z [----] followers, [---] engagements
"@richtrades100 @biopharmacaster Ppmo-51 had Mg problems. Not Eteplirsen. I stand by my statement. dyn51 had clinical hold. Not good for chronic tx. $dyn $srpt"
X Link 2025-12-08T11:58Z [----] followers, [---] engagements
"$dyn safety not as good as $srpt Etep. Srpt also had ppmo51 which was discontinued due to safety and dyne-251 is very close analog. I would scrutinize ongoing chronic safety data very closely. This safety is very mixed and read note 5- ie why did they choose not to report on safety events after 8/19/25"
X Link 2025-12-08T16:10Z [----] followers, [----] engagements
"@BiotechAnalysst Really need a detailed safety breakdown also"
X Link 2025-12-08T18:52Z [----] followers, [---] engagements
"@BiotechAnalysst No new persistent thrombocytopenia for more than [--] months tough for a chronic tx. $dyn $rna $srpt"
X Link 2025-12-08T19:54Z [----] followers, [---] engagements
"@bioinvestor24 Is this correct summary: $dyn dyne-251 produces 7fold increase of D over baseline $rna del-zota produces 4-5fold increase over baseline And according to this $srpt Etep produces 7fold increase over baseline https://pubmed.ncbi.nlm.nih.gov/34120909/#::text=The%20study%20found:%20*%2078/79%20eteplirsen%2Dtreated%20patientsmild%20to%20moderate%20and%20unrelated%20to%20eteplirsen https://pubmed.ncbi.nlm.nih.gov/34120909/#::text=The%20study%20found:%20*%2078/79%20eteplirsen%2Dtreated%20patientsmild%20to%20moderate%20and%20unrelated%20to%20eteplirsen"
X Link 2025-12-09T00:14Z [----] followers, [---] engagements
"$arwr Related to $srpt subQ CNS Ataxias/Huntingtons Disease programs: https://ir.arrowheadpharma.com/static-files/61bc8b9b-ccf4-45d7-85ad-0d11825bdbc2 https://ir.arrowheadpharma.com/static-files/61bc8b9b-ccf4-45d7-85ad-0d11825bdbc2"
X Link 2025-12-10T14:59Z [----] followers, [----] engagements
"@BikeRieder Do you know if this will be owned by $arwr or this is one of the $srpt agreed to programs"
X Link 2025-12-10T16:11Z [----] followers, [---] engagements
"@FirstSquawk lol Nvda knew full well what was going on"
X Link 2025-12-10T18:17Z [----] followers, [--] engagements
"@BioBoyScout @sonichedgefund @RNAiAnalyst Have to imagine $alny would like to grab $arwr (and the stock is trading like they are in play). But otoh I doubt the FTC would allow the deal to go thru"
X Link 2025-12-12T23:52Z [----] followers, [---] engagements
"@sonichedgefund @BioBoyScout @RNAiAnalyst If on off chance $alny interested in $srpt (I would assume the interest would be around the arwr/srpt muscle; CNS assets) - is the deal with srpt structured such that $arwr can block an acquisition by the likes of alny"
X Link 2025-12-12T23:57Z [----] followers, [---] engagements
"@BioBoyScout I havent noticed them on this schedule yet. Do you know exact day and or timeslot $arwr #jpm The #JPM26 schedule is already out. https://t.co/0ZtclKXxdb The #JPM26 schedule is already out. https://t.co/0ZtclKXxdb"
X Link 2025-12-16T21:15Z [----] followers, [---] engagements
"$capr Interesting point- was said [--] of the trial pts were actively on exon skippers. Also Dr McD said even tho most pts were late/borderline ambulatory they did not test for lower limb function. As such thought approval should be consistent with ages in trial (ie dont repeat the mistake $srpt made). Key point for me- in CRL there were manufacturing/CMC issues. Capr has responded on those issues but nothing back from FDA yet"
X Link 2025-12-17T19:37Z [----] followers, [---] engagements
"@biotech_hunter (Contd). Dr McD said they probably would run trial with younger patients. Seems like a no brainer. Also he seemed to indicate not sure if he would give Deramiocel for entire lifetime of patient but would be comfortable for 5+ years at least. $capr"
X Link 2025-12-17T19:40Z [----] followers, [---] engagements
"@biotech_hunter Could be but I simply heard $capr responded and hoped would be sufficient with FDA. But I heard nothing about FDA specific response back. So an open issue for me. Deramiocel is donor derived and need repeat doses so I imagine scale up and standardization is a tricky issue"
X Link 2025-12-17T19:58Z [----] followers, [---] engagements
"@IroquoiInvest $srpt Thats what HHS official said in Newsweek etc: https://www.newsweek.com/hhs-official-newborn-screening-saves-lives-and-empowers-families-opinion-11217094 https://www.newsweek.com/hhs-official-newborn-screening-saves-lives-and-empowers-families-opinion-11217094"
X Link 2025-12-18T20:31Z [----] followers, [----] engagements
"@IroquoiInvest Also great to see Really strong support for these New Born Screenings. VERY Important See all the comments as you scroll down in What They Are Saying link: https://www.hhs.gov/press-room/wtas-secretary-kennedy-adds-duchenne-muscular-dystrophy-metachromatic-leukodystrophy-to-newborn-screenings.html https://www.hhs.gov/press-room/wtas-secretary-kennedy-adds-duchenne-muscular-dystrophy-metachromatic-leukodystrophy-to-newborn-screenings.html"
X Link 2025-12-18T20:38Z [----] followers, [--] engagements
"@JackFarley96 lol think about the markets caps (and insider liquidation events) and debt of much bigger companies that are riding on this $msft $amzn $meta $nvda $avgo $orcl etc etc. SA is literally banking on this"
X Link 2025-12-19T01:55Z [----] followers, [----] engagements
"@RNAiAnalyst Such an insufferable shill 🤡 . God forbid $lly or some other BP have to pay an extra $10 for $abvx"
X Link 2025-12-30T23:39Z [----] followers, [----] engagements
"@obryanjf At least $srpt progress on Endeavor- recruiting again. Cohort [--] (sirolimus) is new of course. Not sure if good sign for LGMD2e BLA filing https://clinicaltrials.gov/study/NCT04626674cond=DMD&term=Endeavor&rank=1&tab=history&a=11&b=12#version-content-panel https://clinicaltrials.gov/study/NCT04626674cond=DMD&term=Endeavor&rank=1&tab=history&a=11&b=12#version-content-panel"
X Link 2026-01-02T18:38Z [----] followers, [---] engagements
"@RNAiAnalyst According to preclinical data Srp-1005 knocks down both WT and mutant HTT- similar to $qure and $ions approaches. $arwr $srpt"
X Link 2026-01-07T17:02Z [----] followers, [---] engagements
"@BioSignal Co-Development So you think the portfolio company will partner in US with someone like Alnylam or Big Pharma"
X Link 2026-01-08T02:43Z [----] followers, [--] engagements
"@MarkC_LVB @ArrowheadPharma Yep here is the spec that APOE could be the next big $arwr CNS program (after PD AD/MAPT $srpt HD): New on Substack (Free): $ARWR APOE on the Menu: What Arrowheads BBB Patent Actually Tells Us A look at the CNS Target X slide APOEs cardiometabolic/CNS biology and Arrowheads new TfR1 BBB patent. How APOE emerges as a clean prototype for a Target Xstyle program and New on Substack (Free): $ARWR APOE on the Menu: What Arrowheads BBB Patent Actually Tells Us A look at the CNS Target X slide APOEs cardiometabolic/CNS biology and Arrowheads new TfR1 BBB patent. How APOE"
X Link 2026-01-09T18:50Z [----] followers, [----] engagements
"And remember as part of $arwr $srpt deal- entered into a discovery collaboration for up to six additional muscle cardiac and/or CNS targets. Would be nice to hear more about those preclinical siRNA programs at JPM. Have seen some interesting targets in their patents: for ex APOE ACVR2 MSTN GYS1. https://twitter.com/i/web/status/2010040530588103102 https://twitter.com/i/web/status/2010040530588103102"
X Link 2026-01-10T17:26Z [----] followers, [----] engagements
"@RNAiAnalyst You may be focused on the wrong companies: see below: $arwr #APOE #AD New on Substack (Free): $ARWR APOE on the Menu: What Arrowheads BBB Patent Actually Tells Us A look at the CNS Target X slide APOEs cardiometabolic/CNS biology and Arrowheads new TfR1 BBB patent. How APOE emerges as a clean prototype for a Target Xstyle program and New on Substack (Free): $ARWR APOE on the Menu: What Arrowheads BBB Patent Actually Tells Us A look at the CNS Target X slide APOEs cardiometabolic/CNS biology and Arrowheads new TfR1 BBB patent. How APOE emerges as a clean prototype for a Target"
X Link 2026-01-10T21:45Z [----] followers, [---] engagements
"@RNAiAnalyst (Contd) $arwr ( $srpt ) #APOE etc: https://patents.google.com/patent/WO2025160275A1/en https://patents.google.com/patent/WO2025160275A1/en"
X Link 2026-01-10T22:25Z [----] followers, [---] engagements
"@Marktillerimark @tickerade Potential targets as part of $arwr $srpt agreement: @RNAiAnalyst (Contd) $arwr ( $srpt ) #APOE etc: https://t.co/09w5Zuj7jd https://t.co/5QyaYAH15L @RNAiAnalyst (Contd) $arwr ( $srpt ) #APOE etc: https://t.co/09w5Zuj7jd https://t.co/5QyaYAH15L"
X Link 2026-01-10T22:35Z [----] followers, [---] engagements
"@Marktillerimark @tickerade Contd $arwr $srpt :"
X Link 2026-01-10T22:48Z [----] followers, [---] engagements
"@BiotechObserver Not sure how you can say most boring ever when there are credible rumors of $30B and $20B advanced BO negotiations. Rumors and media backed spec yes but not boring imo. $rvmd $abvx $mrk $abbv $lly $jnj $azn $xbi #jpm"
X Link 2026-01-13T00:35Z [----] followers, [----] engagements
"@timfris Would not be shocked if $axsm leap frogs both $rvmd / $abvx . $xbi"
X Link 2026-01-14T03:30Z [----] followers, [---] engagements
"Yes I have seen some complaining boring JPM. Investing doesnt necessarily need drama. Look at big tech the last few years- a ton of slow steady multiple expansion. Versus multiple compression in $XBI for years. But weve had huge breakthrus in cancer obesity rare dx etc genetic medicine etc. And now in midst of boring JPM - credible rumors of $20B and $30B BO talks amidst BP patent cliffs. These are the key factors that lead to ongoing multiple expansion once again. https://twitter.com/i/web/status/2011498392698974560 https://twitter.com/i/web/status/2011498392698974560"
X Link 2026-01-14T17:59Z [----] followers, [--] engagements
"@qian_li41394 Hmm did this list Underperform this week"
X Link 2026-01-16T21:08Z [----] followers, [--] engagements
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