Dirk Haussecker

I did not, however, appreciate that acute ApoC3-lowering had such a robust (negative) impact on liver enzymes. It is therefore very comforting to see that they normalize over time, as does HbA1C which had been the primary safety concern. $arwr $ions

$arwr super-impressive: in the 200+ sHTG subjects treated with ApoC3 RNAi #plozasiran and followed for another 2 years in the open-label portion of study....ZERO experienced an acute pancreatitis event. $ions ir.arrowheadpharma.com/static-files/4…

@GeneInvesting It is not just the market realizing the AATD data, but also sickle cell and other data and biz dev. Of course, Trump's narcissism may have put an and to humanity by then, but go bold or go home. $beam

@RNAiAnalyst Wow 5x in a year is bold in this twilight zone of a biotech market. But I can definitely see 3x.

$beam nice to hear resounding positive reaction by analyst community to AATD data. Market reaction was a headscratcher, but those in the know will continue to hold and add if possible. 5x this time next year, especially after they disclose time-relationship of correction.

This would have been a great review if not for the glaring omission of the actual toxic molecular species in Huntington's: exon1a mRNA. It is why seemingly similar knockdown approaches will strikingly differ in their efficacy: $ptct/nvs and $ions/roche should fail (if not worsen HD by increasimg exon1a), while $qure and $alny should and are delivering on their promise. It is the details that matter.

“Huntington’s Disease and The Triad of Therapeutic Conviction”. Debut blog from Eric Green, CEO of Trace Neuro, explaining why and how advances are happening in HD. Genetics, cellular understanding, and enabling modalities. lifescivc.com/2026/03/huntin…

@ShahramSN @RocketPharma @CivilizationVC Congrats. Getting a drug approved for commercialization is a big achievement. Curious, but what was your financial reasoning for backing LAD-I gene therapy given its ultrarare nature? Can it be 'passively commercialized' without it becoming a cash drain? $rckt

Today is a BIG day for @RocketPharma — and for my firm @CivilizationVC!!! An FDA approved drug. 🚀🧬 The FDA granted accelerated approval to Rocket gene therapy for the severe form of a rare immune disorder called leukocyte adhesion deficiency-I (LAD-I). "LAD-I is caused by mutations in the ITGB2 gene and leads the immune system to stop working properly. Patients with severe disease face serious and potentially deadly bacterial and fungal infections. Currently, the only potential cure is stem cell transplant from a donor, but that bears its own serious risks. The gene therapy involves extracting a young patient’s own blood stem cells and modifying them in the lab to introduce functional copies of the gene. Patients then receive conditioning to clear out their bone marrow, after which the modified cells are infused back in hopes of giving them a working immune system." Congratulations to the Rocket team on this milestone! FDA approves Rocket's gene therapy for ultra-rare immune disease - endpoints.news/fda-approves-r…

Still floored by #FDA exploring 'collaboration opportunities' with Arnold Ventures whose main healthcare objective is to reduce expenditures for rare disease drugs. @johnarnold has not only been main financial backer of Prasad's academic work (as well as funding Makary), we now also know that he was involved in FDA staffing decisions and to this month keeps meeting with FDA leadership to influence policy. Time to drain the swamp.

Shivers down the spines of the #rare disease community. On the official calendar: Ex-Enron chief trader #Arnold Ventures continues to meet with its henchmen Makary and Prasad to give instructions.

@ABD1232121 There MUST be a sell-the-news, it's a rule in biotech. See also $dnli approval. $rckt

@RNAiAnalyst Why $rckt is getting down after approval ?!

$rckt after getting its PRV worth $150M+ after 1) much angst around Prasad and uncontrolled trials and 2) in this current environment marked by fears of #FDA delays due to staffing shortages Stock down -7% without even having had a runup prior. OK, now I've seen it all.

Catalyst scorecard: $beam ✔️ $wve 👎 no apparent dose response a disappointment, but market clearly overreacted and tripling my share count in low to mid $5s was right decision $srpt not much data released really, absence of splicing data raised my eyebrows; so no score here (had no position) $rckt ✔️ Any more catalysts I'm right on and I'm broke. $pepg next.

$repl down pre-market despite $rckt approval. Mmh...

$qure when you consider how many Huntington's patients will eventually be on the FDA label and get AMT-130 access, this immediately becomes a drug for an ultrarare population,. Hard to understand why it should be treated differently from $dnli and $rckt ultrarare drugs.

30mg/kg may be a bit more potent, but much quarterly infusions (vs annual subQ) and what about the high discontinuation rate for the 10mg/kg mono group here? What did $lly change to lower it in subsequent cohorts? Also, $lly tested in much higher bmi and diet/exercise requirement (see pbo response). $wve

@RNAiAnalyst bima is far better

Bimagrumab and $arwr tirzepatide combo data clearly show the way how $wve-007 will play out. Traders will want to time for when market wakes up to it (more $wve data needed or before?). I, however, feel pretty waiting for it to play out while enjoying the ADAR and biz development newsflow.

The mental split by an analyst that $wve and $arwr inhibinE RNAi compounds are behaving differently due to some 'idiosyncracy' with wve-007 is something to behold. Of note, this analyst had been a permabear on $arwr until very recently.

$rckt April $5 call option was bid/ask $1.1/1.15 when it was trading at-the-money, implying a roughly 40% move on PDUFA. Right now, stock up barely +14% despite PRV.

$qure considering FDA clearly exercises regulatory flex for ULTRArare genetic diseases...why not take a first step by approving AMT-130 NOW for the small HD subpopulation it was just shown to work in. $rckt $dnli

I returned from Beijing earlier this week, where @Pfizer has been on the ground for 36 years. I heard from our team there about how they're using AI to fundamentally rethink how we develop and deliver medicines, not just incremental improvements but a different way of working entirely. Globally, we intend to be the most AI-forward company in biopharma. This will take the best minds, in every market we serve, working together. That is how breakthroughs happen.