Re $SRPT, huge doses are used, for Elevidys approx 10exp16 genomes for 70kg+, 100x > # of cells in the human body, or an MOI of 100 for every cell the patient has, ie trying to force AAV to do something it's not really capable of: Is it really surprising if tox issues may result?

The $FDMT JPM/Jan25 investor deck is worth a perusal ir.4dmoleculartherapeutics.com/4DMT-Corpora... - finally bringing genetic medicine to large and sustainable markets with actually practical therapies which can really compete with and provide benefits over other modalities - with $506m in cash too

I like these updates from $FDMT today! finance.yahoo.com/news/4dmt-an... finance.yahoo.com/news/4dmt-fo... - focus on high value targets, each with strong clinical data to support the pivotal trials, with a $ runway to 2028 - not just another AAV company in my view

$REPL reports initiation of it's registration-intended randomized controlled Phase 2/3 clinical trial of RP2+nivolumab in CPI naive uveal melanoma, and it's phase 2 clinical trial of RP2+atezo/bev in 2L HCC, ie generally after initial progression on 1L atezo/bev ir.replimune.com/news-release...

To believe "Navacaprant showed an efficacy signal in females" per $NMRA's PR that was actually real (& even that by only one of the measures used), one has to also believe that navacaprant made things actively worse in males by around the same amount: Unlikely I feel ir.neumoratx.com/news-release...

Up 55% since I posted this - jolly good!

How it was, and how it's going, just days apart (and a 50F temperature difference too)

This is clearly nonsense - if 40% of sites had a lower placebo effect, giving this result, then 60% of sites must have had a higher placebo effect, giving the opposite result - math 101 (pls show the results for the other 60%..) $VRTX investors.vrtx.com/static-files...

Intra-vitreal gene therapy for ocular conditions has to be the way to go, ie as compared to subretinal or suprachoroidal injection, even though AAV naturally only achieves quite low transduction efficiency by that route $FDMT finance.yahoo.com/news/4dmt-an...

A nice patient story with $REPL's RP1 (in this case in angiosarcoma) cancer.uiowa.edu/news/iowan-s...

Expecting $CGON's cretostimogene grenadenorepvec to be the first non-HSV-based oncolytic immunotherapy to be FDA approved in the not too distant future..which like T-VEC was constructed (years ago) largely by my ex-PhD student at UCL, Mike Robinson - well done Mike! cgoncology.com/cg-oncology-...

In an age if increasing vaccine skepticism, I like this approach from $CDTX - a once yearly prophylactic antiviral flu shot (not a vaccine, nor dependent on the immune system), which should work for all flu strains and work whatever the immune status of the patient finance.yahoo.com/news/cidara-...

I will certainly be buying this: www.washingtonpost.com/books/2024/1...

Winter has arrived in VT..16F this morning too

$REPL receives Breakthrough Therapy designation for RP1 for advanced melanoma after anti-PD1 & submits BLA to the FDA ir.replimune.com/news-release...

Worth a listen - Sander is great! www.biotechtv.com/post/forbion...

Any pre-revenue biotech paying $100/sqft showed a severe lack of financial discipline in my view, & drinking one's own cool aid, when 10 miles away you can spend 75% less: fitting out a palace in Cambridge just shouldn't be a use for investor's drug development $ www.wsj.com/real-estate/... #biosky

Reality is, the problems are the same as ever for GT- delivery/durability (still only efficient to hardly anywhere), & only a v small # of generally v rare tractable targets; it's always been more a tech looking for an application rather than a broad solution #biosky www.statnews.com/2024/08/22/g...