@RNAiAnalyst
aka Dirk Haussecker. Biotech enthusiast with expertise in sequence-directed therapeutics (RNAi, ASO, ADAR, CRISPR...) enjoying drug development and trying to make a buck along the way.
- #markets these should be 2 quite volatile trading weeks with 2-week-Trump having to decide on Iran and tariffs. Then we could have a little FDA OTP Verdun-firing scare in CGT space on top, or maybe investors have gotten used to it by now.
- Eli Lilly acquires $VERV- official now. finance.yahoo.com/news/lilly-a...
- #CRISPR Stocks in Wake of $VERV Acquisition by Eli Lilly (blog): rnaitherapeutics.blogspot.com/2025/06/cris... $CRSP $LLY $NTLA $BEAM $PRME $MGX $IONS
- #VerveTherapeutics CEO did what was best for the medicine, but also good for shareholders. Though Eli Lilly will get to buy them for too cheap, investors can reinvest quickly into $NTLA $PRME and maybe $MGX for hopefully another easy double. #CRISPR
- #DyneTherapeutics myotonic dystrophy type I regulatory update likely coming tomorrow. These pathway updates have been major biotech stock movers recently. If no big move on day 1, following days usually strong upward momentum. investors.dyne-tx.com/news-release...
- $NTLA-2002 More and More Looks Like a Cure for HAE (blog): rnaitherapeutics.blogspot.com/2025/06/ntla... Jaw-dropping efficacy/safety. Could totally disrupt HAE market, including on-demand. #HAE $IONS $KALV
- #uniQure hires away Kylie O’Keefe from #Huntington's competitor #PTCTherapeutics, planning for AMT-130 launch. Guess she has more confidence in uniQure's ddRNAi over PTC's small molecule reaching market soon. So do I. finance.yahoo.com/news/uniqure...
- What we've learned since #IntelliaTherapeutics liver enzyme disclosure 2 weeks ago: 1) late response (week 4-5)...unlikely LNP tox 2) VERVE-102 uses same ionizable lipid as NTLA-2001/2 (from Novartis) 3) no clinical halts 2 weeks after event #VerveTherapeutics #CRISPR
- Worth repeating: It is unfair to blame the #pharmaceutical industry for the poor #health of US population. It actually makes sense: more $ spent when people are sick. What needs to be addressed is what makes them sick in 1st place: poor nutrition, having to take a car to get to cross a road.
- MAHA Vows to Disrupt Sick-Care Model in Favor of Cures (blog): rnaitherapeutics.blogspot.com/2025/06/maha... #CRISPR #cures #genetherapy #FDA $NTLA $VERV $BBIO 💊
- I bought a German military stock 3 months ago for my kids' future-oriented #portfolio. I checked today: that stock is 80% up. #biotech investing is hard.
- Breaking...2nd Patient with Chronic Granulomatous Disease Successfully Treated with #PrimeEditing (blog): rnaitherapeutics.blogspot.com/2025/06/2nd-... $PRME $BEAM #CRISPR
- #PrimeMedicine CEO said they'll complete dosing of cohort 1 in CGD trial (2-3 patients). I believe this could be enough to get approval under new #FDA. That would be worth ~$150M currently.
- #FDA Dr OZ at #CBER listening session had interesting idea wrt how to pay for $2M Casgevy-type drugs.... #reinsurance, socialize cost so that individual insurance companies do not feel disadvantaged having rare disease patients.
- #FDA HHS secretary Kennedy loves #CRISPR and wants US to be competitive! www.youtube.com/watch?v=x6Lz...
- Another must-watch podcast from the 'new' #FDA. Anybody still in camp that Makary and Prasad are bad for #innovation in drug development seriously needs to re-think. In addition to words, now plenty of action to support: x.com/DrMakaryFDA/...
- #biotech shorts and those that missed the boat by not buying Vinay Prasad appointment are becoming louder...which means we are on the right track. CBER transparency day tomorrow. Important.
- #FDA Sarepta's #rAAVrh74 viral vector in the limb girdle muscular dystrophy program receives #platform designation. This is another sign that 'new' FDA is serious in accelerating rare disease drug development + bodes well for agency's view of recent DMD death: finance.yahoo.com/news/u-fda-g...
- #ProQR votes are out. deBoer (86.3%) + Shannon (80.7%) re-appointed as Directors. Should encourage corp governance discussion. Only 78.5% of votes cast were in favor of authorizing Board to issue shares. Not sure the 'no' are aware that we are in the biotech business? www.sec.gov/Archives/edg...
- #FDA must-read: CBER chief Vinay Prasad wants to speed rare disease patient access to drugs that show any promise, also using surrogate endpoints. Vows not to interfere in review process. My man. endpoints.news/fdas-vinay-p...
- #FDA lots of positive things from Prasad and Makary over last few days. Patient access to medical options is top of agenda. On Thursday, we will hear directly from Prasad. Buckle in. www.fda.gov/vaccines-blo...
- #uniQure this AMT-260 epilepsy n=1 data looks very interesting. If it were an administration procedure artefact, I would have expected the most pronounced effect directly following admin. The opposite is the case.
- #Regeneron is such a cool #genetics-based biotech, but they should also become expert in modalities, not just licensing them. #CRISPR and #RNAediting are ideally suited and they should have them in-house. Prices are cheap, too!
- #RNAi Therapeutics A Bright Spot In Dark Biotech Winter (blog): rnaitherapeutics.blogspot.com/2025/06/rnai... $ALNY $ARWR $SLN $RGLS $QURE
- #VerveTherapeutics and #PrimeMedicine should exchange clinically proven GalNAc-LNP and prime editing rights. Win-win. No need to take undue delivery safety risk here, and #Verve paid too much to #Beam for #CRISPR access.
- #uniQure The 'new' FDA won't stand in the way of a rapid AMT-130 ddRNAi approval for #Huntington's. FDA meeting mintues are back! uniqure.gcs-web.com/news-release...
- Impressive #obesity data from #Regeneron: anti-myostatin antibody + Glp-1 leads to less lean body mass loss, while preserving overall weight loss. Does a matching deal for Glp1/GIP dual with #China Hansoh. investor.regeneron.com/news-release... investor.regeneron.com/news-release...
- Substantial multi-billion $ #biotech dealmakings this morning: #Sanofi acquired Blueprint Medicines for $9B, #BristolMyers in VEGF-A-PD-L1 bispecific 50/50 for essentially $3.5B upfront. finance.yahoo.com/news/biontec... finance.yahoo.com/news/sanofi-...
- #SilenceTherapeutics more p3 #rusfertide polycythemia vera data presented by competitor Protagonist+Takeda at ASCO, incl number of phlebotomies and % <45% hematocrit. #SLN124 compares favorably in earlier studies. finance.yahoo.com/news/protago...
- #IntelliaTherapeutics #CRISPR it looks like tylenol caused gr4 liver enzymes, at least contributed. FDA must pull Tylenol from market, I guess :). x.com/GeneInvestin...
- Pharmaceutical companies should start investing in treatments for vaccine-preventable disease. Should be more lucrative than the #vaccines themselves. #measles #polio jamanetwork.com/journals/jam...
- #RNAi pure-plays #Alnylam and #SilenceTherapeutics continue moving higher. Alnylam soon worth more than #Regeneron. Oligos still much less #China exposure compared to antibodies and CAR-Ts. Me-thinks we'll see eye-cathing M&A soon.
- #ANGPTL3 it may be time for #ArrowheadTherapeutics and #VerveTherapeutics to reposition their ANGPTL3-targeting agents from currently only homoFH and potentially ASCVD...to much larger #MASH opportunity. See also #EliLilly's solbinsiran and Arrowhead's zodasiran ANGPTL3 results:
- #CRISPR what kills me about the 'irreversibility' argument, esp with regard to cancer is that when cancer emerges as a problem with non-permanent modalities, stopping these medicines don't stop the cancer either.
- Today #SummitTherapeutics, last week at ODAC FDA panel rejected STARGLOW (COLUMVI) trial data as not applicable to US population because too many Chinese enrolled. Food for thought for the Big Pharma deal makers rushing to #China for early clinical data.
- #RNAi platform bios #SilenceTherapeutics and #ArrowheadTherapeutics thriving recently. Guess the recent deal flurry in sector is helping. Hope that #Silence has been progressing #inhibinE for obesity as well. That, in addition to p3-ready Lpa, will clinch the deal.
- #ProQR here is the van Herk letter. Makes important points, including violation of Dutch securities laws regarding Director term limits. Annual Meeting next week. www.sec.gov/Archives/edg...
