$XBI $SLDB $SRPT This reminds me of something $SLDB shared recently in a fireside— in dose escalation of their novel DMD gene Tx, which nevertheless bears structural similarities with $SRPT commercial Elevidys, $SLDB “discovered” a region of the gene that should not be disturbed. Fortunately, the effects as to the patient, a young boy, in question were relatively not severe and reversible. However, it could have been much worse, said $SLDB. They reported out their findings to the scientific community, I believe. But shortly thereafter, $SLDB learned that this must-preserve region of the gene was already known to $SRPT, as it was (finally) disclosed in their drug’s label upon regulatory approval…

For medical information, general AI frontier models (Google, OpenAI, Anthropic) outperformed specialized @EvidenceOpen and @UpToDate as assessed by 12 US clinicians, randomized and blinded to which model and extensive testing/benchmarks. This was not anticipated. @NatureMedicine nature.com/articles/s41591-0…

Did we really need another cardiac myosin co? Or another sGC activator fiercebiotech.com/biotech/ca…

Bought the cancer scare meltdown. Planned for a quick swing trade. Now I have a "La Lettre" account.

Your stocks went DOWN because AI stocks went UP So naturally you thought that your stocks would go UP when AI stocks went DOWN! You thought wrong, you dumb bitch!

Base editing in human embryos fixes some mutations and creates others (and creates other problems) article in reply Why is Human Heritable Genome Editing controversial? 1) The science is not ready Embryo editing still has major technical problems, including mosaicism, unintended edits, and uncertainty about long-term effects. 2) The risk is borne by a future person who cannot consent. The edited child has no say in the intervention, and the change may be passed to future generations. 3) There are often safer alternatives. For many inherited diseases, IVF plus preimplantation genetic testing can avoid disease without editing embryos. 4) It creates a pathway from disease prevention to enhancement. The same tools used to prevent disease could be redirected toward traits such as height, cognition, appearance, or perceived “optimization.” h/t @UrnovFyodor for calling the ultrawealthy advocates of HHGE "baby Improvers" 5) It risks privatized eugenics. If genetic “improvement” becomes a service for the wealthy, it could deepen inequality and normalize the idea that some genomes are preferable to others. 6) Governance is weak and jurisdiction-shopping is likely. Even where embryo editing is restricted or banned, private actors may seek permissive jurisdictions, weak oversight, or opaque pathways to proceed. #geneediting #ethics #bioethics

Is Nuvalent just TurningPoint therapeutics again?

Some context for that 92% ORR / 90% 6mth landmark PFS in pancreatic cancer for the $TNGX vopimetostat daraxonrasib combo: vopimetostat monorx 25% ORR, ~90% 6mth PFS $RVMD daraxonrasib monorx 32%, 6mth PFS 56%

$TNGX slides. $RVMD ir.tangotx.com/static-files/…

$JNJ acquiring a pan-KRAS degrader via Firefly Bio for $1B businesswire.com/news/home/2…

$TNGX Tango Therapeutics Announces Combination of Vopimetostat and Daraxonrasib Demonstrated 92% Objective Response Rate in Pancreatic Cancer | Tango Therapeutics, Inc ir.tangotx.com/news-releases…

Yes, small sample size but he’s also making the fallacy of equating equal sample sizes (patient number) to equal AE capture. Sure, the number of patients in the placebo arm was roughly equal to the 50mg arm, but 66% of the placebo arm patients dropped out of the study before completion, versus only 18% of the 50mg arm!!! This means the PATIENT YEAR sample from the placebo arm is likely ~half that of the $ABVX 50mg arm’s patient year sample. So, the placebo arm should naturally be EXPECTED to have a lower rate of cancers, even with the same starting “sample size”, and even with A DRUG THAT DOES NOT HAVE CANCER.

Hopefully the pod bros who sold $ABVX have access to Google or an LLM to learn about what colonic dysplasia and NMSC are. Wealth transfer to the specialists in process.

$ABVX Rare to get a full 6.5 hour trading day to pull the trigger like this. With marginal understanding of UC market, stats, and how cancers develop it took maybe 2 hours of work to get comfortable. Sheep, Adam, others right, price driving narrative. (In both directions)

Nice review of current and emerging literature in CRC Given so much emphasis on low ORRs in late line mCRC, interesting to hear MD quite enthusiastic for 10% delta on OS at 24mos and lower incidence of HFMD for zanzalitnib Relavent $EXEL, perhaps $CTMX open.spotify.com/episode/1QQ…