# ![@Hall8Jack Avatar](https://lunarcrush.com/gi/w:26/cr:twitter::2598246906.png) @Hall8Jack Jack Hall

Jack Hall posts on X about $srpt, $arwr, $rna, $nvs the most. They currently have [-----] followers and [---] posts still getting attention that total [---] engagements in the last [--] hours.

### Engagements: [---] [#](/creator/twitter::2598246906/interactions)
![Engagements Line Chart](https://lunarcrush.com/gi/w:600/cr:twitter::2598246906/c:line/m:interactions.svg)

- [--] Week [---------] +88,649%
- [--] Month [---------] +44,162%
- [--] Months [---------] +8,743%
- [--] Year [---------] +3,835%

### Mentions: [--] [#](/creator/twitter::2598246906/posts_active)
![Mentions Line Chart](https://lunarcrush.com/gi/w:600/cr:twitter::2598246906/c:line/m:posts_active.svg)

- [--] Week [--] -40%
- [--] Month [--] +119%
- [--] Months [---] +96%
- [--] Year [---] +43%

### Followers: [-----] [#](/creator/twitter::2598246906/followers)
![Followers Line Chart](https://lunarcrush.com/gi/w:600/cr:twitter::2598246906/c:line/m:followers.svg)

- [--] Week [-----] -0.19%
- [--] Month [-----] -0.50%
- [--] Months [-----] +4.10%
- [--] Year [-----] +9.50%

### CreatorRank: [---------] [#](/creator/twitter::2598246906/influencer_rank)
![CreatorRank Line Chart](https://lunarcrush.com/gi/w:600/cr:twitter::2598246906/c:line/m:influencer_rank.svg)

### Social Influence

**Social category influence**
[stocks](/list/stocks)  70.59% [finance](/list/finance)  7.56% [countries](/list/countries)  5.04% [celebrities](/list/celebrities)  0.84% [technology brands](/list/technology-brands)  0.84%

**Social topic influence**
[$srpt](/topic/$srpt) #18, [$arwr](/topic/$arwr) 39.5%, [$rna](/topic/$rna) 37.82%, [$nvs](/topic/$nvs) 15.97%, [$rhhby](/topic/$rhhby) #4, [$dyn](/topic/$dyn) 12.61%, [$pepg](/topic/$pepg) 8.4%, [$qure](/topic/$qure) 6.72%, [$alny](/topic/$alny) 5.88%, [$rgnx](/topic/$rgnx) 3.36%

**Top accounts mentioned or mentioned by**
[@ttramyloid](/creator/undefined) [@sanctuarybio](/creator/undefined) [@rnaianalyst](/creator/undefined) [@jamesmclaw70633](/creator/undefined) [@yachmod](/creator/undefined) [@seedy19tron](/creator/undefined) [@bikerieder](/creator/undefined) [@tanavoi373](/creator/undefined) [@elmonogran42994](/creator/undefined) [@towiu2](/creator/undefined) [@persimmonti](/creator/undefined) [@arrowheadpharma](/creator/undefined) [@mrdarkghost](/creator/undefined) [@biotechobserver](/creator/undefined) [@richtreonze](/creator/undefined) [@sonichedgefund](/creator/undefined) [@prosperousguy](/creator/undefined) [@monacobiotech](/creator/undefined) [@sarepta](/creator/undefined) [@gantosj](/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) [uniQure N.V. (QURE)](/topic/$qure) [Alnylam Pharmaceuticals, Inc. (ALNY)](/topic/$alny) [Eli Lilly and Company (LLY)](/topic/$lly) [Viking Therapeutics, Inc (VKTX)](/topic/$vktx) [Wave Life Sciences Ltd. Ordinary Shares (WVE)](/topic/$wve) [Taysha Gene Therapies, Inc. (TSHA)](/topic/$tsha) [Intra-Cellular Therapies Inc. (ITCI)](/topic/$itci) [Insmed, Inc. (INSM)](/topic/$insm) [Amgen, Inc. (AMGN)](/topic/$amgn) [GSK plc (GSK)](/topic/$gsk) [Entrada Therapeutics, Inc. (TRDA)](/topic/$trda) [Pfizer, Inc. (PFE)](/topic/$pfe) [Metsera, Inc. (MTSR)](/topic/$mtsr) [Structure Therapeutics Inc. (GPCR)](/topic/$gpcr) [Amphenol Corporation (APH)](/topic/$aph) [ArcelorMittal (MT)](/topic/$mt) [CannabisCoin (CANN)](/topic/cannabis)
### Top Social Posts
Top posts by engagements in the last [--] hours

"$rna $dyn $pepg $arwr / $srpt etc: $RNA myotonic dystrophy p3 results had been expected in Q2 now likely in Sep/Oct. Wouldn't surprise me if $DYN will have very similar endpoints. https://t.co/nmgYpdKTGR $RNA myotonic dystrophy p3 results had been expected in Q2 now likely in Sep/Oct. Wouldn't surprise me if $DYN will have very similar endpoints. https://t.co/nmgYpdKTGR"  
[X Link](https://x.com/Hall8Jack/status/2014472464844112289)  2026-01-22T22:57Z [----] followers, [---] engagements


"I agree could be; Medicines Co is a very good baseline analogy. But beyond that the question for me is is how much RNAi IP does $srpt have (via $arwr ) for muscle etc: Sarepta has an exclusive license to Arrowheads technology to develop therapeutics against a broad range of skeletal muscle gene targets. https://twitter.com/i/web/status/2016580631807885610 https://twitter.com/i/web/status/2016580631807885610"  
[X Link](https://x.com/Hall8Jack/status/2016580631807885610)  2026-01-28T18:34Z [----] followers, [---] engagements


"@BikeRieder @sonichedgefund @Bionerd51 So $srpt paid $arwr . IMO good for all involved . Look frwd to data (and remember a subQ arm was added late last year- could be a Key differentiator with $rna $dyn $pepg )"  
[X Link](https://x.com/Hall8Jack/status/2016825697587626446)  2026-01-29T10:48Z [----] followers, [---] engagements


"@Investorclimber Already have precedence of kidney tox at 10mg/kg in DMD (note clinical holdS). Does not bode great $pepg : https://www.fiercebiotech.com/biotech/pepgen-ends-all-work-duchenne-muscular-dystrophy-after-lead-asset-fails-raise-dystrophin https://www.fiercebiotech.com/biotech/pepgen-ends-all-work-duchenne-muscular-dystrophy-after-lead-asset-fails-raise-dystrophin"  
[X Link](https://x.com/Hall8Jack/status/2017113179369635963)  2026-01-30T05:50Z [----] followers, [---] engagements


"@prosperousguy @ElMonoGran42994 @monaco_biotech Potentially a big deal as $srpt / $arwr will try to deliver subcutaneous vs brain surgery for $qure . Most patients would obviously much prefer subQ over brain surgery. SubQ in a CNS disease would be a huge breakthrough #HD #siRNA"  
[X Link](https://x.com/Hall8Jack/status/2019060031690256589)  2026-02-04T14:46Z [----] followers, [----] engagements


"@prosperousguy @ElMonoGran42994 @monaco_biotech (Contd) but I hope both $qure $srpt eventually get approved for HD"  
[X Link](https://x.com/Hall8Jack/status/2019060410507317291)  2026-02-04T14:48Z [----] followers, [---] engagements


"@jonesallen99138 $srpt $arwr Which study would you guess will publish CNS PK/PD/engagement data first INSIGHTT/Study SRP-1005-101 or AROMAPT-SC-1001 https://clinicaltrials.gov/study/NCT07221344term=Arrowhead&sort=StudyFirstPostDate&limit=10&page=1&rank=4 https://clinicaltrials.gov/study/NCT07221344term=Arrowhead&sort=StudyFirstPostDate&limit=10&page=1&rank=4"  
[X Link](https://x.com/Hall8Jack/status/2019213953994486030)  2026-02-05T00:58Z [----] followers, [----] engagements


"How does this bode for $qure HD AMT-130 $rgnx $RGNX gets FDA CRL for RGX-121 BLA in Hunter syndrome (MPS II) - just missing the Feb [--] PDUFA. Issues cited: neuronopathic patient population definition natural history external control comparability and surrogate endpoint validity (CSF HS D2S6). Company plans Type A $RGNX gets FDA CRL for RGX-121 BLA in Hunter syndrome (MPS II) - just missing the Feb [--] PDUFA. Issues cited: neuronopathic patient population definition natural history external control comparability and surrogate endpoint validity (CSF HS D2S6). Company plans Type A"  
[X Link](https://x.com/Hall8Jack/status/2021012083761103170)  2026-02-10T00:03Z [----] followers, [----] engagements


"RT @RoKhanna: .@matthewschmitz believes that my coining the phrase Epstein class & @ossoff using it subsequently in a speech is "antisemiti"  
[X Link](https://x.com/Hall8Jack/status/2022434569111179611)  2026-02-13T22:15Z [----] followers, [---] engagements


"@RNAiAnalyst When you look at it from that perspective- the only thing that makes sense is stock market (volatility) manipulation. Which probably explains why boy wonder was fired in the first place. And when they realize they got away with it they brought him back $xbi $ibb"  
[X Link](https://x.com/Hall8Jack/status/2022482417119105323)  2026-02-14T01:25Z [----] followers, [---] engagements


"@RNAiAnalyst Does this mean $rna / $nvs struggling to hit stat sig If so wouldnt be first time in muscular dystrophy obviously"  
[X Link](https://x.com/Hall8Jack/status/2014473451214422249)  2026-01-22T23:01Z [----] followers, [---] engagements


"@TOWiU2 (Contd) so another interesting Elevidys dynamic- $rhhby $srpt combined will do $260M in 4Q25. That is about the same run rate as Zolgensma ($300M 3Q25 and which $nvs purchased for $9B in 2018)"  
[X Link](https://x.com/Hall8Jack/status/2018351849502363904)  2026-02-02T15:52Z [----] followers, [---] engagements


"RT @JMaraganore: Insane"  
[X Link](https://x.com/Hall8Jack/status/2022426470497554658)  2026-02-13T21:43Z [----] followers, [--] engagements


"@TOWiU2 I believe thats the single best quarter of revenue $rhhby has had for Elevidys and more than $srpt had in US for Q4. Interesting dynamic"  
[X Link](https://x.com/Hall8Jack/status/2018265566834798815)  2026-02-02T10:09Z [----] followers, [---] engagements


"Biospace and others want you to believe $rgnx has a nextgen to $srpt Elevidys. At the same time rgnx has a clinical hold AND CRL. My guess is the data will show rgnx-202 is nothing more than me too. $sldb too The FDA raised concerns about its clinical trials ability to properly define a patient population the use of a natural history control arm and the use of a biomarker as a surrogate endpoint FDA rejects Regenxbio's gene therapy in Hunter syndrome https://t.co/cjFzcoBoSO The FDA raised concerns about its clinical trials ability to properly define a patient population the use of a natural"  
[X Link](https://x.com/Hall8Jack/status/2021015727814172963)  2026-02-10T00:17Z [----] followers, [----] engagements


"@Sports_BioTech1 $dtil $lly : https://www.fiercebiotech.com/biotech/lillys-prevail-walks-away-gene-editing-collab-precision https://www.fiercebiotech.com/biotech/lillys-prevail-walks-away-gene-editing-collab-precision"  
[X Link](https://x.com/Hall8Jack/status/2021577734682472595)  2026-02-11T13:31Z [----] followers, [---] engagements


"Looks like $2M list price in Japan for $rhhby / Chugais Elevidys. Covered by insurance. $srpt"  
[X Link](https://x.com/Hall8Jack/status/2022269894822137919)  2026-02-13T11:21Z [----] followers, [---] engagements


"RT @jakescottMD: Important thread from @matthewherper on the data FDA refused to review. Worth noting: FDA reviewed Moderna's trial desig"  
[X Link](https://x.com/Hall8Jack/status/2022482660296147062)  2026-02-14T01:26Z [----] followers, [--] engagements


"RT @PersimmonTI: $xbi @wsj the pressure to make a move rises back to business means back to rules & predictability $qure reversal $mrn"  
[X Link](https://x.com/Hall8Jack/status/2022526415636468151)  2026-02-14T04:20Z [----] followers, [--] engagements


"@yachmod @Sarepta $srpt has the Emergene expression data but they still need Voyagene expression functional safety data for BLA. So imo no. See bottom slide:"  
[X Link](https://x.com/Hall8Jack/status/1945929321409048970)  2025-07-17T19:31Z [----] followers, [---] engagements


"Not many talk about these [--] trials but are they now fairly critical for DMD GTx and $srpt $rgnx $sldb etc going forward Could make very big difference. Have read that rgnx and sldb have sig higher Ab exclusions than srpt: https://clinicaltrials.gov/study/NCT06241950cond=DMD&term=Sarepta%20&page=3&rank=21 https://clinicaltrials.gov/study/NCT06597656cond=DMD&term=Sarepta%20&page=2&rank=14 https://clinicaltrials.gov/study/NCT06241950cond=DMD&term=Sarepta%20&page=3&rank=21 https://clinicaltrials.gov/study/NCT06597656cond=DMD&term=Sarepta%20&page=2&rank=14"  
[X Link](https://x.com/Hall8Jack/status/1945939623055638751)  2025-07-17T20:12Z [----] followers, [---] engagements


"@RNAiAnalyst @JamesMclaw70633 SRP-1005 MOA is lowering the huntingtin (HTT) protein. It does not target an exon. So can you explain why targeting exon [--] transcript is critical $srpt $arwr $alny $qure"  
[X Link](https://x.com/Hall8Jack/status/1945963067180740942)  2025-07-17T21:45Z [----] followers, [----] engagements


"@RNAiAnalyst @JamesMclaw70633 Right but $arwr slide says targets Mutant (key) and WT. So is it possible that it is better than targeting Exon [--] transcript alone IDK"  
[X Link](https://x.com/Hall8Jack/status/1945969179376447819)  2025-07-17T22:09Z [----] followers, [---] engagements


"@steveusdin1 @InvestrClouseau For whatever its worth dosing was complete by early April [----] for [----] P1 study. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-pipeline-progress-multiple-limb https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-pipeline-progress-multiple-limb"  
[X Link](https://x.com/Hall8Jack/status/1946023057170042940)  2025-07-18T01:43Z [----] followers, [---] engagements


"@LBR_ZA Do you have a link for the Roche claim @shindaejin0722 Roche already halted Elevidys orders. The pivot to siRNA is underway lower risk recurring revenue strong [--------] outlook. The 90% institutional stat Unverifiable. No fund holds through -60%. Current narratives are totally detached from reality. #SRPT #RNAi #Biotech @shindaejin0722 Roche already halted Elevidys orders. The pivot to siRNA is underway lower risk recurring revenue strong [--------] outlook. The 90% institutional stat Unverifiable. No fund holds through -60%. Current narratives are totally detached from reality. #SRPT"  
[X Link](https://x.com/Hall8Jack/status/1946718834187997439)  2025-07-19T23:48Z [----] followers, [---] engagements


"@RNAiAnalyst $arwr DM1 does not want a brain penetrant Ab delivery mechanism. Huntingtons does. So two different delivery mechanisms. Compare their smaller muscle dose (DM1/FSHD) compared to $rna / $dyn approach: https://ir.arrowheadpharma.com/static-files/c24adf87-2bec-4068-9d55-38da5058c5c9 https://ir.arrowheadpharma.com/static-files/c8100bbd-963d-4275-9233-c0b3f18f6d31 https://ir.arrowheadpharma.com/static-files/c24adf87-2bec-4068-9d55-38da5058c5c9 https://ir.arrowheadpharma.com/static-files/c8100bbd-963d-4275-9233-c0b3f18f6d31"  
[X Link](https://x.com/Hall8Jack/status/1947401014249984136)  2025-07-21T20:59Z [----] followers, [---] engagements


"@Stmkrs @Sanctuary_Bio @HealthcareNinj1 The deaths were all in NA. The full approval and submitted Embark P3 data were based on ambulatory"  
[X Link](https://x.com/Hall8Jack/status/1947711491991605670)  2025-07-22T17:33Z [----] followers, [--] engagements


"$rhhby $srpt (1/3) https://www.reuters.com/sustainability/boards-policy-regulation/roche-pauses-shipments-elevidys-gene-therapy-outside-us-2025-07-22/ https://www.reuters.com/sustainability/boards-policy-regulation/roche-pauses-shipments-elevidys-gene-therapy-outside-us-2025-07-22/"  
[X Link](https://x.com/Hall8Jack/status/1947830958842855544)  2025-07-23T01:27Z [----] followers, [---] engagements


"(3/3) .local reg authorities. Elevidys has been approved by regulatory authorities in [--] Roche territories for the treatment of DMD - Bahrain Brazil Israel Japan Kuwait Oman Qatar and the UAE. So which of these reference FDA as the basis for their local approval"  
[X Link](https://x.com/Hall8Jack/status/1947833261742301186)  2025-07-23T01:36Z [----] followers, [---] engagements


"$srpt $rhhby Updated: In a follow-up conversation on the safety study the senior official said Sarepta could make a change to the dosing or manufacturing and test it on the next [--] or [--] boys and show the liver-related issues were improved Updated: In a follow-up conversation on the safety study the senior official said Sarepta could make a change to the dosing or manufacturing and test it on the next [--] or [--] boys and show the liver-related issues were improved"  
[X Link](https://x.com/Hall8Jack/status/1948462684497674458)  2025-07-24T19:17Z [----] followers, [---] engagements


"So is it a dosing problem or manufacturing problem Very confusing. Would make sense if it was a few bad batches issue. That could be very fixable. Pressure would be on both $srpt $rhhby and the contract manufacturer"  
[X Link](https://x.com/Hall8Jack/status/1948464438832710019)  2025-07-24T19:24Z [----] followers, [----] engagements


"@BioTecIsFuture Absolutely crazy . What are the chances $rhhby stepped in today to be the voice of reason If so the $srpt ceo needs to go"  
[X Link](https://x.com/Hall8Jack/status/1948484655545758186)  2025-07-24T20:45Z [----] followers, [---] engagements


"$rhhby $srpt :"  
[X Link](https://x.com/Hall8Jack/status/1948702571712954673)  2025-07-25T11:11Z [----] followers, [----] engagements


"@PersimmonTI $srpt $rhhby Probably not but wonder if this is related (timing interesting) Just in: Roche $RHHBY confirms that the death of a Duchenne muscular dystrophy patient in Brazil treated with Sarepta's $SRPT Elevidys is unrelated to the gene therapy according to Bloomberg. The patient was not part of a clinical trial. Just in: Roche $RHHBY confirms that the death of a Duchenne muscular dystrophy patient in Brazil treated with Sarepta's $SRPT Elevidys is unrelated to the gene therapy according to Bloomberg. The patient was not part of a clinical trial"  
[X Link](https://x.com/Hall8Jack/status/1948864282193801485)  2025-07-25T21:53Z [----] followers, [----] engagements


"@PersimmonTI $srpt $rhhby https://www.tipranks.com/news/the-fly/roche-says-brazilian-death-unrelated-to-elevidys-bloomberg-reports-thefly https://www.tipranks.com/news/the-fly/roche-says-brazilian-death-unrelated-to-elevidys-bloomberg-reports-thefly"  
[X Link](https://x.com/Hall8Jack/status/1948866275247030596)  2025-07-25T22:01Z [----] followers, [---] engagements


"$rhhby $srpt I'm told this is the case from Brazil. Regulators there determined the boy likely died from flu intensified by immunosuppression https://t.co/oP3PE6fxJa I'm told this is the case from Brazil. Regulators there determined the boy likely died from flu intensified by immunosuppression https://t.co/oP3PE6fxJa"  
[X Link](https://x.com/Hall8Jack/status/1948868273677681032)  2025-07-25T22:09Z [----] followers, [---] engagements


"$rhhby $srpt UPDATE 1-FDA probes death of patient on Sarepta's Elevidys partner Roche says death unrelated to therapy https://t.co/bgakZWKPm3 UPDATE 1-FDA probes death of patient on Sarepta's Elevidys partner Roche says death unrelated to therapy https://t.co/bgakZWKPm3"  
[X Link](https://x.com/Hall8Jack/status/1948887962424672263)  2025-07-25T23:27Z [----] followers, [---] engagements


"@Gantosj Cant help but wildly speculate. Timing of Brazil incident (flu) and news of DM1 $arwr program dose escalating. One can bash $srpt all you want (much v deserved) but otoh one can argue they have done more for muscular dystrophy community than all other biotechs combined (1/2)"  
[X Link](https://x.com/Hall8Jack/status/1949934741748142203)  2025-07-28T20:47Z [----] followers, [----] engagements


"@Gantosj (2/2) so I cant help but speculate key fda official/muscular dystrophy community see big pic that there is rationale for right to try for Elevidys (no deaths YET in [---] amb pts) AND there is desire for v important $arwr $srpt DM1 AND FSHD clinical trials to move frwd smoothly"  
[X Link](https://x.com/Hall8Jack/status/1949936825373507909)  2025-07-28T20:55Z [----] followers, [----] engagements


"@MSollender On off chance this is really true this points to a manufacturing issue/glitch no If so just have to hope easily fixable $srpt $rhhby #Catalent"  
[X Link](https://x.com/Hall8Jack/status/1949942961975747065)  2025-07-28T21:20Z [----] followers, [---] engagements


"@BAMBossie81 Likely not but nevertheless is an interesting point: Timing of FDA negativity (compared to when they first were informed of fatalities) and Stat non stop over the top neg articles compared to timing of CHMP opinion. $rhhby $srpt"  
[X Link](https://x.com/Hall8Jack/status/1949945049850007743)  2025-07-28T21:28Z [----] followers, [---] engagements


"@RxRegA Or some jumped to overly simple conclusions (narratives) during a ridiculously complicated/nuanced series of events "  
[X Link](https://x.com/Hall8Jack/status/1949988092749677027)  2025-07-29T00:19Z [----] followers, [--] engagements


"@KavodCapital Sounds like Marks may have known about this $srpt $rhhby Did they not see that even their ally Peter Marks said the pause makes sense per @maxonwifi reporting from earlier today. Also Marks: It is possible that the manufacturing complexity and dosing issues are larger than just this one program." https://t.co/BDS9Xf0ybf How do Did they not see that even their ally Peter Marks said the pause makes sense per @maxonwifi reporting from earlier today. Also Marks: It is possible that the manufacturing complexity and dosing issues are larger than just this one program.""  
[X Link](https://x.com/Hall8Jack/status/1950012990142443727)  2025-07-29T01:58Z [----] followers, [----] engagements


"@3IDWarrant @Alnylam @ArrowheadPharma Well if it embraced just 1/2 as much that is a ten bagger. lol. $50B mc $alny vs $2B mc $arwr "  
[X Link](https://x.com/Hall8Jack/status/1950928719280808326)  2025-07-31T14:37Z [----] followers, [---] engagements


"@kevinnbass You realize that the same Dr. Mendell of Nationwide Childrens Hospital that invented Zolgensma (owned by Novartis) for SMA also invented Elevidys IMO very difficult to find any other individual who has done more for SMA DMD and LGMD than Dr. Mendell (and his colleagues at NCH)"  
[X Link](https://x.com/Hall8Jack/status/1951735147746746717)  2025-08-02T20:01Z [----] followers, [---] engagements


"@FidelisAurelius This is factually untrue. Dr. Mendell of Nationwide Childrens Hospital invented Elevidys before AA due to biomarkers was law. The same time he invented Zolgensma which is a proven successful Tx for SMA. Many years after his inventions Sarepta and Novartis licensed these Txs"  
[X Link](https://x.com/Hall8Jack/status/1951743238009950360)  2025-08-02T20:33Z [----] followers, [--] engagements


"@ShortReportMeta @MartinShkreli Yes despite mixed to negative FAERS news- $srpt trading more in line today with $arwr $alny (siRNA). The future value for srpt going forward is clearly siRNA (DM1 FSHD SCA HD; IPF ). Could be trial update with data soon for DM1 FSHD and/or IPF. All with large TAMs"  
[X Link](https://x.com/Hall8Jack/status/1952434006248534521)  2025-08-04T18:18Z [----] followers, [---] engagements


"@Sanctuary_Bio @seedy19tron (1/2) Little bit surprised $srpt not up more on $nvs $rna BO rumor. IMO most of the value in Avidity is in DM1; FSHD (better data than $dyne altho safety signals/clinical holds for both in neuromuscular). Sarepta behind time wise but supposed have much smaller dose than Avidity : $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 [--] 🧐"  
[X Link](https://x.com/Hall8Jack/status/1953208824086610340)  2025-08-06T21:37Z [----] followers, [---] engagements


"@Sanctuary_Bio @seedy19tron (2/2) For more discussion/implications of supposed smaller $arwr $srpt dose in DM1/FSHD (compared to $rna) listen to Arrowhead RBC presentation on 5/20 (key discussion starts at half way point). And look at these slides: 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/1953210158353461725)  2025-08-06T21:42Z [----] followers, [---] engagements


"@Sanctuary_Bio @seedy19tron (3/3) One other thing to note - $srpt likely looked at $rna and $dyn before they did the $arwr deal (DM1 FSHD SCAs HD):"  
[X Link](https://x.com/Hall8Jack/status/1953213048359141673)  2025-08-06T21:54Z [----] followers, [---] engagements


"@Sanctuary_Bio @seedy19tron And remember $rna had clinical hold in DM1 so smaller $arwr $srpt dose could be very pertinent : https://www.cureduchenne.org/wp-content/uploads/2022/09/Avidity-Biosciences-Announces-FDA-Partial-Clinical-Hold-on-New-Participant-Enrollment-in-Phase-1.pdf https://www.cureduchenne.org/wp-content/uploads/2022/09/Avidity-Biosciences-Announces-FDA-Partial-Clinical-Hold-on-New-Participant-Enrollment-in-Phase-1.pdf"  
[X Link](https://x.com/Hall8Jack/status/1953215164494303732)  2025-08-06T22:02Z [----] followers, [---] engagements


"@Sanctuary_Bio @seedy19tron FSHD has 20k patients. DM1 40k patients. Total unmet need. $alny trades at 10x [----] sales. If $rna really has the siRNA goods in DM1/FSHD they should NOT sell out for $7-10B . More like $14B+ like $itci . Just mo"  
[X Link](https://x.com/Hall8Jack/status/1953234444388880490)  2025-08-06T23:19Z [----] followers, [---] engagements


"@RNAiAnalyst Do you know what if anything critical differentiates $arwr INHBE vs $wve INHBE (siRNA)"  
[X Link](https://x.com/Hall8Jack/status/1953530585130942684)  2025-08-07T18:56Z [----] followers, [---] engagements


"@scartissue123 $arwr dimer : https://ir.arrowheadpharma.com/static-files/c15dcdf5-1930-4472-8511-5fb4b5e9eab9 https://ir.arrowheadpharma.com/static-files/c15dcdf5-1930-4472-8511-5fb4b5e9eab9"  
[X Link](https://x.com/Hall8Jack/status/1953684370574705009)  2025-08-08T05:07Z [----] followers, [---] engagements


"@scartissue123 $arwr dimer contd :"  
[X Link](https://x.com/Hall8Jack/status/1953684824222302478)  2025-08-08T05:08Z [----] followers, [---] engagements


"@WonderExplorer2 They set up the plans late last year. Data was well known. Some sold more than 1/2 their shares. If you were clearly best in class in DM1/FSHD1 (huge unmet need) would you really plan to dump 50% or more of your shares; $15M $rna $dyn $arwr"  
[X Link](https://x.com/Hall8Jack/status/1954977467200049594)  2025-08-11T18:45Z [----] followers, [----] engagements


"@WonderExplorer2 $rna very well could. But if I were $nvs I would wait for $arwr data this year to set price of BO. DM1/FSHD big enough for multiple siRNA drugs imo"  
[X Link](https://x.com/Hall8Jack/status/1954984369707168192)  2025-08-11T19:12Z [----] followers, [----] engagements


"@TTRAmyloid Agree with what you say about the DM1/FSHD market opportunity but there are caveats. 1) the data is fairly mediocre: (1/2) $rna $dyn $pepg $arwr $srpt 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/1956053761052266781)  2025-08-14T18:02Z [----] followers, [---] engagements


"@TTRAmyloid (2/2) And 2) there is this: $rna $dyn $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 potentially allows $SRPT to toggle to quarterly subQ administration vs every 6-8 week IV) Good"  
[X Link](https://x.com/Hall8Jack/status/1956054361559237066)  2025-08-14T18:04Z [----] followers, [----] engagements


"$rna insiders continue to take advantage of $nvs rumor. $dyn $pepg $arwr $srpt"  
[X Link](https://x.com/Hall8Jack/status/1956478237980213583)  2025-08-15T22:08Z [----] followers, [---] engagements


"@JamesMclaw70633 @tanavoi373 $arwr just updated INHBE FSHD DM1 Ataxia etc trials also. Full steam ahead. Interesting after initial $rna spike on $nvs rumor- arwr trending strongly up; rna trending down. If look at overlap (DM1/FSHD $srpt ) and breadth of their pipelines and relative mcs- makes sense imo"  
[X Link](https://x.com/Hall8Jack/status/1958204448594968972)  2025-08-20T16:28Z [----] followers, [---] engagements


"@JamesMclaw70633 @tanavoi373 Not to mention recent obesity stumbles by $lly $vktx opens the door for companies with different MoAs from glp1 like $arwr $wve etc etc"  
[X Link](https://x.com/Hall8Jack/status/1958206740597711347)  2025-08-20T16:37Z [----] followers, [---] engagements


"@JamesMclaw70633 @tanavoi373 Lot of dynamics ( $alny $rna / $nvs $lly / $vktx ) potentially in $arwr favor currently. Next they just need to deliver more clinical data wins (after Plozasiran)"  
[X Link](https://x.com/Hall8Jack/status/1958214646428561761)  2025-08-20T17:08Z [----] followers, [--] engagements


"@JamesMclaw70633 @tanavoi373 Lot of dynamics ( $alny $rna / $nvs $lly / $vktx ) potentially in $arwr favor currently. Next they just need to deliver more clinical data wins (after Plozasiran Zodasiran Olpasiran Fazirsiran)"  
[X Link](https://x.com/Hall8Jack/status/1958223937336221849)  2025-08-20T17:45Z [----] followers, [---] engagements


"$srpt $rhhby $rgnx $sldb $insm etc. : BREAKING NEWS: @MDAorg and @ParentProjectMD have published Consensus Guidelines for safe equitable delivery of #GeneTherapy in #Duchenne #MuscularDystrophy Read more https://t.co/RDO0Wi66mX https://t.co/teeno3iNuh BREAKING NEWS: @MDAorg and @ParentProjectMD have published Consensus Guidelines for safe equitable delivery of #GeneTherapy in #Duchenne #MuscularDystrophy Read more https://t.co/RDO0Wi66mX https://t.co/teeno3iNuh"  
[X Link](https://x.com/Hall8Jack/status/1959455834041380948)  2025-08-24T03:20Z [----] followers, [----] engagements


"$arwr $nvs . $nvs $rna $dyn $srpt $pepg etc : Wow: $ARWR and $NVS Enter into a Global License and Collaboration Agreement - $200M upfront https://t.co/CMKaBfNb4p Wow: $ARWR and $NVS Enter into a Global License and Collaboration Agreement - $200M upfront https://t.co/CMKaBfNb4p"  
[X Link](https://x.com/Hall8Jack/status/1962883971148116104)  2025-09-02T14:23Z [----] followers, [---] engagements


"Related thread $nvs $arwr $rna $srpt : @TTRAmyloid (2/2) And 2) there is this: $rna $dyn $arwr $srpt @TTRAmyloid (2/2) And 2) there is this: $rna $dyn $arwr $srpt"  
[X Link](https://x.com/Hall8Jack/status/1962885166348509329)  2025-09-02T14:27Z [----] followers, [---] engagements


"$nvs $arwr $RNA (Avidity insiders active lately): https://app.quotemedia.com/data/downloadFilingwebmasterId=90423&ref=319423883&type=PDF&symbol=RNA&cdn=4b9027ccdc776300e67744ab701c1512&companyName=Avidity+Biosciences+Inc.&formType=4&formDescription=Statement+of+changes+in+beneficial+ownership+of+securities&dateFiled=2025-08-29"  
[X Link](https://x.com/Hall8Jack/status/1962888325934534917)  2025-09-02T14:40Z [----] followers, [---] engagements


"@yaireinhorn @BiomedicalRX @ArrowheadPharma $arwr has now signed CNS deals with both $nvs and $srpt (arwr/srpt deal also included Muscle targets). Srpt at that time either passed on ARO-SNCA for PD or could not afford it"  
[X Link](https://x.com/Hall8Jack/status/1962892644180259005)  2025-09-02T14:57Z [----] followers, [---] engagements


"@yaireinhorn @BiomedicalRX @ArrowheadPharma In terms of siRNA interest- with this $arwr deal will $nvs start to sniff around $srpt in addition to $rna (DM1/FSHD1)"  
[X Link](https://x.com/Hall8Jack/status/1962894568984027462)  2025-09-02T15:05Z [----] followers, [---] engagements


"@MrDarkghost Less than a month ago (siRNA) $nvs $rna $arwr $srpt (DM1/FSHD1/DMD): Novartis $NVS Weighs Deal For Biotech Avidity $RNA To Boost Drug Pipeline - FT @mroliverbarnes @JFK_America @hannahkuchler 🧬Novartis has been evaluating a bid for Avidity and expressed interest in an acquisition in recent weeks. 🧬Avidity is working with advisers to assess https://t.co/Yhmt4F89m3 Novartis $NVS Weighs Deal For Biotech Avidity $RNA To Boost Drug Pipeline - FT @mroliverbarnes @JFK_America @hannahkuchler 🧬Novartis has been evaluating a bid for Avidity and expressed interest in an acquisition in"  
[X Link](https://x.com/Hall8Jack/status/1962896965550678109)  2025-09-02T15:14Z [----] followers, [----] engagements


"@MrDarkghost $arwr $srpt deal- Muscle (DM1 FSHD etc) plus CNS (3 Ataxias Huntingtons Disease etc). Srpt also had a look at PD and AD at that time but not part of deal"  
[X Link](https://x.com/Hall8Jack/status/1962917952941863269)  2025-09-02T16:38Z [----] followers, [---] engagements


"@commonsenseplay @MrDarkghost I personally think if somebody hits clearly best in class (thru 2030) siRNA assets in DM1 and FSHD its worth ITCI buyout type money ($14B)"  
[X Link](https://x.com/Hall8Jack/status/1962922571235487853)  2025-09-02T16:56Z [----] followers, [--] engagements


"@a_a_free One small problem with $arwr is they are partnering away most revenue opportunities. So have to also look at who will own Most of their best siRNA assets. $nvs $srpt $amgn $tak $gsk etc"  
[X Link](https://x.com/Hall8Jack/status/1962926188583702748)  2025-09-02T17:10Z [----] followers, [---] engagements


"@a_a_free And of course big question is can another siRNA company like $arwr start to close the huge valuation gap with $alny ($60B) at some point $rna $srpt $dyn etc"  
[X Link](https://x.com/Hall8Jack/status/1962928587465494705)  2025-09-02T17:20Z [----] followers, [---] engagements


"@TTRAmyloid Interesting while $nvs was supposedly closing a BO of $rna - instead they partner with $arwr in PD. Both big siRNA players. Is Novartis going all in on siRNA or did they have a change of heart on best available siRNA assets Arwr/ $srpt vs Avidity (DM1/FSHD)"  
[X Link](https://x.com/Hall8Jack/status/1962983071239831941)  2025-09-02T20:56Z [----] followers, [---] engagements


"@TTRAmyloid @bioinvestor24 Will $rna hit stat sig in P3 Might not be slam dunk 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/1962984462112731562)  2025-09-02T21:02Z [----] followers, [---] engagements


"@edcollins23 @MauriceOnTW @Sanctuary_Bio @BiotechObserver My guess is Elevidys GNT0004 RGX202 SGT003 will all have similar functional data from their P3 trials. Then as you stated in this thread safety will largely be dependent on Ab titer exclusions. My question is does $srpt still have US rights to GNT0004 https://www.genethon.com/genethon-pursues-different-strategies-for-ensuring-patient-access-to-gene-therapies-for-rare-diseases/ https://www.genethon.com/genethon-pursues-different-strategies-for-ensuring-patient-access-to-gene-therapies-for-rare-diseases/"  
[X Link](https://x.com/Hall8Jack/status/1963354818799444023)  2025-09-03T21:34Z [----] followers, [---] engagements


"@edcollins23 @yachmod @MauriceOnTW @Sanctuary_Bio @BiotechObserver If DI had half a brain he should re partner asap. I doubt Genethon has any effective ability to commercialize outside of EU"  
[X Link](https://x.com/Hall8Jack/status/1963372532515401969)  2025-09-03T22:44Z [----] followers, [---] engagements


"@markflowchatter Probably nothing but $rna insiders have sold $20M triggered since $nvs BO rumors. Latest- Chief Commercial Officer sold 2/3 of his shares over last month: https://app.quotemedia.com/data/downloadFilingwebmasterId=90423&ref=319431429&type=PDF&symbol=RNA&cdn=0b24233cb7ec1fc1a4373bce81f9db8c&companyName=Avidity+Biosciences+Inc.&formType=144&formDescription=Notice+of+proposed+sale+of+securities+pursuant+to+Rule+144&dateFiled=2025-09-03"  
[X Link](https://x.com/Hall8Jack/status/1963397541967376823)  2025-09-04T00:23Z [----] followers, [---] engagements


"@Sanctuary_Bio I think 2D has the biggest TAM of their [--] lead LGMD GTxs. Still sad - the combined actions of FDA journalists (unnamed FDA sources lol) and of course Srpt likely (unnecessarily) damaged the progress of these GTxs that the (LG) MD community were/are keenly interested in"  
[X Link](https://x.com/Hall8Jack/status/1963613660502229227)  2025-09-04T14:42Z [----] followers, [---] engagements


"@TTRAmyloid @BearBullDebate @adamfeuerstein Exactly if $rna takes [--] that would say good chance they think share DM1/FSHD1 market with someone else. And in fact may not be BIC. $arwr $srpt $dyn 👀 ($20M in insider sales this past month)"  
[X Link](https://x.com/Hall8Jack/status/1963636788674662553)  2025-09-04T16:14Z [----] followers, [---] engagements


"@JoseRestonVA Looks like multiple insiders sold more than half their shares this past month according to multiple Form 4s If so tough to characterize as non event. But could be. https://app.quotemedia.com/data/downloadFilingwebmasterId=90423&ref=319362162&type=PDF&symbol=RNA&companyName=Avidity+Biosciences+Inc.&formType=4&formDescription=Statement+of+changes+in+beneficial+ownership+of+securities&dateFiled=2025-08-08"  
[X Link](https://x.com/Hall8Jack/status/1963642118066364600)  2025-09-04T16:35Z [----] followers, [---] engagements


"@Sanctuary_Bio @BiotechObserver Remember its after the CEO failed to disclose that one death in the restructuring PR : after that the FDA put the hammer down on srpt. But they knew the details far before that. Which is fascinating in and of itself"  
[X Link](https://x.com/Hall8Jack/status/1963658782367760791)  2025-09-04T17:41Z [----] followers, [--] engagements


"@richtreonze @TTRAmyloid $rna dose limiting tox details: https://www.neurologylive.com/view/fda-removes-partial-hold-myotonic-dystrophy-agent-aoc-1001 https://www.fiercebiotech.com/biotech/fda-slaps-partial-hold-aviditys-lead-program-docking-dystrophy-study-over-serious-adverse https://www.neurologylive.com/view/fda-removes-partial-hold-myotonic-dystrophy-agent-aoc-1001 https://www.fiercebiotech.com/biotech/fda-slaps-partial-hold-aviditys-lead-program-docking-dystrophy-study-over-serious-adverse"  
[X Link](https://x.com/Hall8Jack/status/1963820833408717036)  2025-09-05T04:25Z [----] followers, [---] engagements


"@richtreonze @TTRAmyloid $rna dose limited to [--] mg/kg in DM1/FSHD (didnt get to [--] mg/kg) $arwr planning to look at [--] and [--] mg/kg in their DM1/FSHD studies: I believe part [--] of dose escalation has begun so we will see if they get there. https://ir.arrowheadpharma.com/news-releases/news-release-details/arrowhead-pharmaceuticals-earns-100-million-milestone-sarepta https://ir.arrowheadpharma.com/static-files/c8100bbd-963d-4275-9233-c0b3f18f6d31 https://ir.arrowheadpharma.com/news-releases/news-release-details/arrowhead-pharmaceuticals-earns-100-million-milestone-sarepta"  
[X Link](https://x.com/Hall8Jack/status/1963853366938796338)  2025-09-05T06:35Z [----] followers, [---] engagements


"@JoseRestonVA @richtreonze @TTRAmyloid Not short. Think $rna has decent shot at approvals. And think $nvs may be looking hard at acquiring them. But nvs should wait for competitor (ie $arwr) data to see if best in class. Broken record- if have clearly BIC siRNA DM1/FSHD data- worth ITCI type BO $ ($14B). $dyn $pepg"  
[X Link](https://x.com/Hall8Jack/status/1963854782101086570)  2025-09-05T06:40Z [----] followers, [---] engagements


"@7to34 @petenajarian @jonnajarian @MarketRebels @RevShark Yet $rna insiders (including ceo) continue selling. Think over $25M in last month : https://app.quotemedia.com/data/downloadFilingwebmasterId=90423&ref=319437076&type=PDF&symbol=RNA&cdn=5d52e7a86bc435fefdeb6d761fb82c0f&companyName=Avidity+Biosciences+Inc.&formType=144&formDescription=Notice+of+proposed+sale+of+securities+pursuant+to+Rule+144&dateFiled=2025-09-05"  
[X Link](https://x.com/Hall8Jack/status/1964119347082723778)  2025-09-06T00:11Z [----] followers, [---] engagements


"@TTRAmyloid I look forward to $rna functional data. Key point- Elevidys reduces CK by a similar amount in placebo trials. So I suppose that means it is healing muscle also (despite your claim it is useless):"  
[X Link](https://x.com/Hall8Jack/status/1964495191190229032)  2025-09-07T01:05Z [----] followers, [---] engagements


"@TTRAmyloid (Contd) In terms of muscle healing will be interesting to compare eventual $rna MRI data to $srpt Elevidys MRI data : This could be key as I believe FDA used MRI data to support Duvyzat approval: https://globalgenes.org/raredaily/fda-approves-italfarmacos-duvyzat-for-dmd/ https://investorrelations.sarepta.com/static-files/c48ffacc-12e0-4592-a9cf-791dff73990e https://globalgenes.org/raredaily/fda-approves-italfarmacos-duvyzat-for-dmd/ https://investorrelations.sarepta.com/static-files/c48ffacc-12e0-4592-a9cf-791dff73990e"  
[X Link](https://x.com/Hall8Jack/status/1964506431232831698)  2025-09-07T01:50Z [----] followers, [---] engagements


"@TTRAmyloid Your comparison of $rna aoc1044 vs $srpt exondys51 is likely very misleading as these exons represent different disease states-ie their baseline levels of D are very different. For ex. aoc1044 produces only a [---] fold increase of D over baseline (contd): https://cureduchenne.org/research/avidity-biosciences-announces-positive-aoc-1044-data/ https://cureduchenne.org/research/avidity-biosciences-announces-positive-aoc-1044-data/"  
[X Link](https://x.com/Hall8Jack/status/1964540280755146841)  2025-09-07T04:04Z [----] followers, [---] engagements


"@TTRAmyloid While $srpt exondys51 produces a [---] fold increase of D over baseline. In terms of ea companies tech you should mention this in above $rna analysis imo as the more relevant ratio is [---] vs 2.8; not the 30:1 you mention (which is clearly apples to oranges): https://www.exondys51.com/what-is-exondys51/about https://www.exondys51.com/what-is-exondys51/about"  
[X Link](https://x.com/Hall8Jack/status/1964545207753679188)  2025-09-07T04:24Z [----] followers, [----] engagements


"@TTRAmyloid Also worth mentioning exon [--] is roughly 6% of all DMD. Obviously a very important treatment but small TAM. $rna #ns $trda $srpt $dyn $pepg etc. me too tech"  
[X Link](https://x.com/Hall8Jack/status/1964548257721512164)  2025-09-07T04:36Z [----] followers, [---] engagements


"@TTRAmyloid (2/2 contd) And $rna had safety signals (ie a discontinuation) for a Chronic treatment. Keeping in mind exon [--] is 6% of DMD so 1k patients $srpt 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/1965778745878589830)  2025-09-10T14:05Z [----] followers, [----] engagements


"@BikeRieder Bodes well for $srpt too:"  
[X Link](https://x.com/Hall8Jack/status/1965884453424910420)  2025-09-10T21:05Z [----] followers, [---] engagements


"@Sanctuary_Bio @bio_clouseau Kind of shareholder hostile- $rna didnt deny BO rumors while insiders dumped $25M the last month . But used rumors to raise at elevated prices. Good smart (if not manipulative) raise imo; they are hedging ahead of their P3s (2 discontinuations/ok efficacy) and competitor data"  
[X Link](https://x.com/Hall8Jack/status/1966132031928050062)  2025-09-11T13:29Z [----] followers, [---] engagements


"@TTRAmyloid Agree good raise. Interesting as $rna insiders keep selling every week since BO rumor; hasnt stopped even with raise: https://app.quotemedia.com/data/downloadFilingwebmasterId=90423&ref=319446848&type=PDF&symbol=RNA&cdn=b8303d616164872e565af80d8d3f479b&companyName=Avidity+Biosciences+Inc.&formType=144&formDescription=Notice+of+proposed+sale+of+securities+pursuant+to+Rule+144&dateFiled=2025-09-11"  
[X Link](https://x.com/Hall8Jack/status/1966498277932593496)  2025-09-12T13:44Z [----] followers, [---] engagements


"@scamsweeper Agree $rna BO potentially on table but suitors are probably waiting for competitive data due very soon from $arwr and $pepg . I believe avidity saw dose limiting tox that limited them to [--] mg/kg; both Pepg and Arwr have gotten beyond that dose in their current P1/2 studies (1/2)"  
[X Link](https://x.com/Hall8Jack/status/1966540632450560328)  2025-09-12T16:33Z [----] followers, [---] engagements


"@IroquoiInvest Agreed this is a very key trial on many levels (EU and newborn screening) $srpt $rhhby: 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/1967683397951910261)  2025-09-15T20:14Z [----] followers, [---] engagements


"@yachmod @JoshInvestValue @Sarepta DM1 and FSHD siRNA $arwr data could very well be reported here $srpt : https://www.wms2025.com/ https://www.wms2025.com/"  
[X Link](https://x.com/Hall8Jack/status/1967686018984972634)  2025-09-15T20:24Z [----] followers, [----] engagements


"@TTRAmyloid There are less than 1k x44 patients. Small TAM. DelZota has had a discontinuation in small N trial so there are worrisome safety signals. Also was a discontinuation in $rna DM1 program. And the data isnt even clearly better let alone 10x better lol @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): https://t.co/9E6QdetPDd Also: https://t.co/EgYWoZjUTO @TTRAmyloid To look at actual data $rna Del Zota data seems pretty similar to $srpt Elevidys vs natural history In fact this"  
[X Link](https://x.com/Hall8Jack/status/1968014091878011370)  2025-09-16T18:08Z [----] followers, [---] engagements


"@TTRAmyloid Also interesting it was inferred $nvs would BO $rna and instead nvs did a partnership with $arwr. Followed by rna diluted shareholders instead of securing rumored BO . lol #AvidityRugPull"  
[X Link](https://x.com/Hall8Jack/status/1968843502151704607)  2025-09-19T01:04Z [----] followers, [----] engagements


"@doepke_michel $rhhby $srpt Elevidys [----] pts treated total. Now compare to very small N $rna x44 nat history (EC) data- very similar efficacy. One time vs chronic "  
[X Link](https://x.com/Hall8Jack/status/1970166961855431030)  2025-09-22T16:43Z [----] followers, [---] engagements


"$pfe $mtsr highly differentiated. $arwr #replicate #haya $gpcr #superluminal #septerna etc etc. #Obesity"  
[X Link](https://x.com/Hall8Jack/status/1970180905462288556)  2025-09-22T17:38Z [----] followers, [---] engagements


"@avidresearch Lot of variables here. CNS vs muscle. Different types of administration and different endpoints. But yes the keys are full length protein vs truncated; the real key is TAMs. HD is big TAM but AMT-130 requires brain surgery- big commercial unknown and competitive txs $qure $srpt"  
[X Link](https://x.com/Hall8Jack/status/1970857349876437471)  2025-09-24T14:26Z [----] followers, [---] engagements


"@blippd @scamsweeper Early data- $pepg clearly better splicing than $rna at single dose; rna saw DLT at 4mg/kg. Pepg has gotten beyond 4mg/kg but need to see MAD studies- that is very key. Pepg has had multiple clinical holds (1/2 contd): https://www.biospace.com/fda/deja-vu-for-pepgen-as-fda-places-hold-on-dmd-drug https://www.biospace.com/fda/deja-vu-for-pepgen-as-fda-places-hold-on-dmd-drug"  
[X Link](https://x.com/Hall8Jack/status/1970957725640372726)  2025-09-24T21:05Z [----] followers, [----] engagements


"@RNAiAnalyst $arwr $srpt HD IND was supposed to be filed before end of [----]. Still opportunity to change RNA sequence if need be"  
[X Link](https://x.com/Hall8Jack/status/1971364224627839399)  2025-09-26T00:00Z [----] followers, [---] engagements


"@JamesMclaw70633 @RNAiAnalyst Great point as I believe $arwr $srpt siRNA approach is NOT intrathecal for HD as opposed to other RNA approaches; subQ instead"  
[X Link](https://x.com/Hall8Jack/status/1971572038708936976)  2025-09-26T13:46Z [----] followers, [---] engagements


"@yachmod I never saw that Chile approved $rhhby $srpt Elevidys. Do you know how this patient qualified to receive treatment"  
[X Link](https://x.com/Hall8Jack/status/1971585268315783401)  2025-09-26T14:38Z [----] followers, [---] engagements


"@Sanctuary_Bio How could a veteran prof at Stanford do something so blockheaded .Unless it is motivated by securities/market manipulation Could very likely be the legacy of this fda leadership given precedent of other recent blockheaded moves (ie unnamed sources etc). $xbi"  
[X Link](https://x.com/Hall8Jack/status/1972808512616247797)  2025-09-29T23:39Z [----] followers, [---] engagements


"All of a sudden investors are strongly embracing natural history/ matched control data in Gene Therapy/RNA therapy $tsha (Rett) $qure (HD) $rna (x44 DMD) etc after mocking $srpt (DMD) etc similar data in the past. What does fda really think 🙏🏼 $xbi"  
[X Link](https://x.com/Hall8Jack/status/1973751293639589968)  2025-10-02T14:05Z [----] followers, [---] engagements


"@cureffi Ahh I just saw AMT-130 lowers both. So I assume Arrowhead approach with subQ delivery/supposedly good brain delivery is a very rational approach"  
[X Link](https://x.com/Hall8Jack/status/1976016286133178690)  2025-10-08T20:06Z [----] followers, [---] engagements


"@richtrades100 If you look at recent pps performance and regulatory updates $tsha and $ngne have gotten for their Rett GTx or $qure for their HD GTx- $srpt *should* have very similar path for their LGMD2e GTx"  
[X Link](https://x.com/Hall8Jack/status/1976817749554086176)  2025-10-11T01:10Z [----] followers, [---] engagements


"$cgc $twmjf $aph $mt estimated at $7billion market - Demand Will Far Outstrip Supply For Legal Canadian Cannabis via http://rbl.ms/1Yw7ciU http://rbl.ms/1Yw7ciU"  
[X Link](https://x.com/Hall8Jack/status/679129506951634945)  2015-12-22T02:41Z [----] followers, [--] engagements


"#exondys51 #EMA #NICE $SRPT https://x.com/mduk_news/status/838086193287954433 If you or your child could benefit from #Duchenne drug #Exondys51 then help us send evidence to the EMA. More info https://t.co/coGcEGAB7Z https://t.co/epPYZhMnv7 https://x.com/mduk_news/status/838086193287954433 If you or your child could benefit from #Duchenne drug #Exondys51 then help us send evidence to the EMA. More info https://t.co/coGcEGAB7Z https://t.co/epPYZhMnv7"  
[X Link](https://x.com/Hall8Jack/status/838736788546027522)  2017-03-06T13:03Z [----] followers, [--] engagements


"Found on Wiley website. Looks like $SRPT PPMO that will go in human clinical trials soon will be published shortly"  
[X Link](https://x.com/Hall8Jack/status/902191196289851393)  2017-08-28T15:28Z [----] followers, [--] engagements


"$SRPT partner Prof Dickson/Genethon to present their DMD microD gene therapy tomorrow. https://www.esgct.eu/Content/pdf/Main_congress_091017.pdf https://www.esgct.eu/Content/pdf/Main_congress_091017.pdf"  
[X Link](https://x.com/Hall8Jack/status/920367216419328002)  2017-10-17T19:13Z [----] followers, [--] engagements


"$avxs $srpt $abeo #NationwideChildrensHospital http://pediatricsnationwide.org/2018/04/02/viruses-that-treat-gene-therapy-comes-of-age/ http://pediatricsnationwide.org/2018/04/02/viruses-that-treat-gene-therapy-comes-of-age/"  
[X Link](https://x.com/Hall8Jack/status/982638055147343877)  2018-04-07T15:15Z [----] followers, [--] engagements


"$AVXS $ABEO $SRPT #Myonexus http://pediatricsnationwide.org/2018/04/02/viruses-that-treat-gene-therapy-comes-of-age/ http://pediatricsnationwide.org/2018/04/02/viruses-that-treat-gene-therapy-comes-of-age/"  
[X Link](https://x.com/Hall8Jack/status/993240118524891136)  2018-05-06T21:24Z [----] followers, [--] engagements

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