
Occam's Toothbrush
378 posts

Occam's Toothbrush
@occams_tooth
Biotech Operator | Not investment advice




Tessera posts 85% blood cell editing data, teasing an off-the-shelf fix for sickle cell disease. endpoints.news/tessera-reveal…

$RGNX Duchenne gene therapy study results are out: microdystrophin expression reached an average of 71% of normal across all 31 boys at 12 wks. Functional improvements in a subset of boys followed for one year also seen. The big question remains: Will the FDA still follow the $SRPT precedent and grant accelerated approval to the Regenxbio gene therapy? CEO Curran Simpson told me the company is holding off on submitting to the FDA until early next year. “I think our data checks every single box that you would want for accelerated approval,” he told me. "We’re not in a rush to go to the FDA at this point. We want to give time for the leadership changes to occur. But that’s OK, because we can still meet our timeline of securing an approval in 2027." More details in our story, written with @Jasonmmast in the following post.


NEW - Trump has signed off on a plan to fire his FDA Commissioner, Dr. Marty Makary. gift link below

Next-gen Duchenne drug from $TRDA disappoints Startup was competing against $DYNE, $NVS and others to develop new exon skippers and other muscle-targeted drugs statnews.com/2026/05/07/ent…



We’ve entered a strategic partnership with @MadrigalPharma to advance an RNAi therapeutic for MASH, combining the precision of Arrowhead’s TRiM™ platform with Madrigal’s liver disease expertise. Read the full announcement: bit.ly/42SoWza

First randomized trial to show Ozempic reduces alcohol consumption in people seeking treatment for alcohol use disorder. Placebo-controlled, double-blind. Participants with BMI >30 kg/m2. thelancet.com/journals/lance… @TheLancet

Well over a billion dollars raised for $ORKA’s decade+ late psoriasis me-too drugs. I can’t emphasize enough the fact there are ZERO psoriasis patients under my care that we cannot control with existing options. 1-2 shots per year instead of 4 is not meaningful innovation, period. Over a billion dollars going to this instead of other medical innovation. Psoriasis is just about the *only* condition in my field where we flat out don’t need something new, and these funds are just stuffing cash down ORKA’s throat to get their performance locks the easy way instead of investing somewhere that could actually benefit someone. As long as nobody sells for a while they’ll all make money, even if these stupid companies can’t get an exit in the long run. The investing system *should* be set up such that it doesn’t reward this nonsense - so far it isn’t. I know there are a lot of smart people involved in this who I otherwise like and respect, but I won’t mince words (hopefully they’ll at least respect me for that): This entire YTE situation is a pathetic use of the incentive system that’s supposed to ultimately help patients by creating medical innovation. As investors we are nothing but parasites if we aren’t taking risks to profit off of innovation and financial engineering our way to success instead. The only way this YTE situation works in the long run from these current valuations is if they are able to exploit a completely broken payor system by forcing doctors to abandon perfectly good generic drugs that the system could cheaply afford. I think this whole thing is gross.

Flagship's new startup Serif Biomedicines launched with $50M to crack non-viral gene therapy delivery, a challenge that has sunk multiple predecessors. endpoints.news/flagships-late…

Venrock landed a 45x return on Kelonia -- turned $20M into $900M, @ky_lahucik + I learned. Could grow to 100x if the milestones hit. endpoints.news/kelonia-invest…


Paragon launched its 7th company today -- Korsana Biosciences, merging into the $CYCN public shell. New ticker will be $KRSA. Lead drug candidate is a shuttled anti-amyloid beta antibody for Alzheimer's disease in early studies. ir.cyclerion.com/news-releases/…













