Dirk Haussecker
@RNAiAnalyst
Biotech enthusiast in full MABA mode, grown up with the RNAi story. Trading focus on nucleic acid-based and -targeted bio. http://rnaitherapeutics.blogspot.com
More on the man destroying @POTUS legacy for helping patients. FDA’s Vinay Prasad Stands with Progressive Health Policies realclearhealth.com/articles/2025/…
$arwr showing some after-hours strength on back of $srpt win.
I have a suggestion: 'promote' Vinay Prasad to 'Chief Digitization Officer' (scanning/digitizing old papers in some underground vault at #FDA).
Amazing. @US_FDA has 800 million pieces of paper in storage from the last 100 years — a goldmine of medical information. Enjoyed visiting our warehouse staff this morning to talk about digitizing and modernizing.
$SRPT great win! #FDA (=Prasad) in self-preservation mode. Claims that removal of hold due to finding that death of Brazilian boy unrelated to Elevidys. May I remind them (=Prasad) that they demanded hold because of LGMD death, not Brazilian case? 🤡
Here is FDA commish @DrMakaryFDA defending #Prasad ('not a political bone in his body'; 'staff love him'). Let's see how long he will keep it up.
FDA commissioner @MartyMakary defends Chief Medical and Scientific Officer Vinay Prasad from criticism, calling him an “impeccable scientist." He talked to our @DashaBurns about how partisan politics interact with his role on this week’s episode of #TheConversation. Subscribe…
$MGX running away from me...I wasn't done adding!!! 😡 Wednesday $IONS earnings. Maybe updates there? We may be underestimating their tech prowess. Would $IONS go for old-style Cas9 or rather base editing?
$ARWR $SRPT what does today's news about timing of (1st) ARO-DM1 data? p1/2 started dosing March 2024 single and mult-dose ascending in DM1 patients July '25: 1st enrollment milestone met 2nd enrollment milestone expected by year-end --> today single-dose enrollment completed?
Arrowhead has reached a key milestone in our collaboration with Sarepta Therapeutics: the first enrollment target in the Phase 1/2 study of ARO-DM1 for type 1 myotonic dystrophy. Learn more: bit.ly/4or9y6P
Copy-cat hunting down innovator #Verona/Merck based on quick p1 POC in China. Go get 'em.
Another major China deal to start the week. GSK and Hengrui. $500M up front. Up to 12 programs, including a PDE3/4 inhibitor. prnewswire.com/news-releases/…
EU-US trade deal: 15% tariff also on #pharmaceuticals. Better than 200% I guess, but dont be surprised if things change again.
Together, the EU and the US are a market of 800 million people. And nearly 44 percent of global GDP. It’s the biggest trade deal ever ↓
That's exactly what happened. On the 91st day after the infusion, Pedro went to school and was infected with the H1N1 virus and died.
Hey @Sarepta + @ArrowheadPharma, y not target by #RNAi MHC Class I presentation genes B2M, TAP1/TAP2 in hepatocytes to moderate T-cell responses against AAV capsid epitopes? Should be easy enough, no, and the entire systemic AAV gene therapy industry may adopt? $srpt $arwr
Vinay Pasad's approach to regulatory decision making: if I dont believe in the underlying concept, the studies that suggest otherwise must be flawed and therefore the drug must be rejected. No need to look at data in more detail. I can do that in 2 hours on PDUFA day querying…
Hey, @DrMakaryFDA, why not get rid of all FDA scientific reviewers since Vinay is over-ruling them in the end anyway? But didnt you say that this would not happen under your watch. What changed? #FDA
$srpt the Brazilian boy died less than 3 months after receiving Elevidys due to flu. During this time, he was likely still on precautionary immunosuppression given as part of gene therapy administration. Not because of liver injury caused by elevidys. academiadepacientes.com.br/a-breve-espera…
Vinay #Prasad's Days as Head of CBER May Be Numbered (blog): rnaitherapeutics.blogspot.com/2025/07/vinay-… $srpt #elevidys #FDA #genetherapy #AAV
What if science could completely change the treatment of cardiovascular disease—from chronic care to a one-and-done treatment? Today we officially acquired Verve Therapeutics, expanding our pipeline of cardiometabolic health medicines. e.lilly/4kTgoP9
$srpt Doug scores another blow to Vinay.
Yes, DMD children on corticosteroids face heightened flu mortality risk due to immunosuppression, which exacerbates their existing respiratory muscle weakness. Sources like Parent Project MD note steroids increase infection susceptibility, while a 2019 meta-analysis links them to…
Hey @grok, are kids with Duchenne's more susceptible to die from flu?
A week gone by without a #Prasad FDA Direct podcast appearance. Eill ee ever see him again?