RazorSharpDNA 🧬

Plenty in Biotech, more of a 5 year bet though. $RXRX $ABCL $HALO $NTLA $CRSP $CERT $DNA $RLAY $SANA $BEAM

1/🚨WOW! @BeamTx has presented initial clinical data from its ongoing Phase 1/2 clinical trial evaluating $BEAM - 301 - a liver-targeting lipid-nanoparticle (LNP) formulation of adenine base editing (ABE) editors designed to correct the R83C mutation, as a “one and done” treatment for patients with glycogen storage disease type Ia (GSDIa). The most significant finding in today’s clinical data was that in the 40 mg cohort, two patients have achieved 15 hours of controlled fasting, improvements in glucose control and no longer required starch supplementation following a single administration of BEAM-301. These results strengthen BEAM-301’s potential as a “one and done” Gene Editing treatment for GSDIa patients by replacing the current intensive daily management which is currently required for treating this disease. Another strong indication of Base Editing’s potential as a meaningful therapeutic modality.

Solid 3 Year Hold Time Frame Large Cap #Stock list. Only 1 #Biotech on the list, but IMO it’s a 💎 so good pick $GMAB Genmab 👀 ⬇️

$IOVA Reports Updated IOV-LUN-202 Results and Adds Precision TIL Strategy for Second-Line mNSCLC 24.6% Objective Response Rate (ORR) in 69 Patients with Non-Squamous mNSCLC ir.iovance.com/news-releases…

$RAPP $175M offering priced at $21/share 😬

Surprise 1: Anthropic acquired a one-person biotech . . . Surprise 2: that person is me Surprise 3: I’m here to tell you all about it Yes, this is what you think it is; after 6 yrs of increasingly loose anonymity: the Professor Oak reveal post …and yes, it has gotten so boring around here that I’m literally doxxing myself to spice up the timeline ------- Ok, allow me to introduce myself. My name is Cody Tranbarger. I mostly tweet, but occasionally find time to dabble in drug dev & starting/building biotechs. My past lives span the VC, crossover, startup continuum; Prof Oak has been my way of keeping a digital foot in those worlds, despite having my physical feet planted elsewhere. The past 18 mo, ‘elsewhere’ was Mithril Medicine, a co. I founded to develop bsAbs for LAMA2-CMD & other ECM-related genetic diseases. Mithril was born out of hands-on building with @MikeNGladstone, @JasonPaulRhodes, @LifeSciVC, & team @atlasventure. Somehow, I made enough half-decent calls along the way (even a broken clock is right twice a day, eh?) to convince them Mithril/I warranted a small seed. While I can hardly rationalize such an outrageously cavalier bet, I’m grateful for it, & I'd partner with Atlas again in a heartbeat. In typical AV fashion, Mithril’s primordial form was virtual, decentralized, & ultra-lean. This stage tends to be short-lived, but for Mithril, it worked surprisingly well, for surprisingly long, thanks in part to the rapid maturation of the AI tools I used liberally. Eventually, Mithril became a bit of an experiment itself - to find the upper bound of how far it could scale as a single operator + a bunch of Claude (+ a stellar bench of part-time consultants). In parallel, I started spending time with @nc_frey, @an1lam, & @CoefficientBio team; and promptly experienced, for the 1st time, AI at the frontier. Soon thereafter, Mithril stopped looking like an experiment, & started looking like a preview of an entirely new operating model. From that spark came “Pipeline-in-a-Person.” The idea immediately resonated when we shared it here back in April; and it was central to the shared vision that led to Anthropic’s acquisition of Mithril, & my joining the team. Why join Anthropic? Three reasons. (1) For Mithril, the programs continue, with more resources & infrastructure behind them, and as much Claude as I can put to work. (2) For me, the chance to start several more programs, & find out how many one person can actually run in parallel - an almost unprecedented luxury for a curious drug hunter. (3) For our mission, a path to institutionalize & scale this new way of working, then put it in the hands of all of biopharma, all over the world - which, candidly, I find the most energizing. Will AI x Bio cure diseases? Unequivocally yes, imo… but not by the route that gets the most airtime. Rather than timeline acceleration & compression, I see orchestration & parallelization as today's low-hanging fruit. Most drug hunters see a handful of go/no-go calls in a career, & spend entirely too much of the rest trading emails (my inbox can attest). Give them the bandwidth to run several programs at once, and the time to focus on scientific judgment, and I’m willing to bet they’ll make better calls. And at scale, better calls translate to more drugs, higher hit rates, broader patient impact, & likely, a disease or 10 or 100 cured. I couldn't be more excited to build toward that future @AnthropicAI. Finally, a heartfelt thank you to BioTwitter/X. The end of anonymity is bittersweet, but if anyone’s earned a proper introduction, you all have. It’s difficult to overstate how much this app has done for me: a decade of education, plenty of friends, many job opportunities, several mentors, a few investors, even a co-founder (fmr). It was the generosity of this community that inspired me to start posting 6 years ago, after just as many years lurking. I hope I’ve been able to pay it forward, in my own small way. But, to be clear, Prof Oak isn’t going anywhere. Same beat, same themes, and now, a front-row seat to drug development at the frontier. Stay tuned for dispatches from the trenches; and remember... Gotta Cure 'Em All

$VNDA Vanda Pharmaceuticals — Phase 3 NEREUS (tradipitant) hit its primary endpoint, any vomiting after semaglutide fell to 43.0% vs 58.5% on placebo, a 27% cut — Vomiting with severe nausea down 35%, no new safety signals — The 60% cut in frequent vomiting and 81% cut in extreme vomiting are post hoc analyses — Plans to meet the FDA and file an sNDA for GLP-1 related vomiting prevention — Risk flag: single 1 mg semaglutide dose, no gradual titration like real use Not financial advice. My earlier VNDA tweet had this wrong. The 27% to 81% was not one range, 27% was the primary result and 81% was the post hoc extreme vomiting number.