Robert Coffin

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

4DMT Announces Positive Interim Data from 4D-150 SPECTRA Clinical Trial in DME and Alignment with FDA on Registrational Path Across all DME patients dosed to date, 4D-150 continues to be well tolerated with no intraocular inflammation observed at any timepoint or dose level3E10 vg/eye demonstrated strong signals of clinical...

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...

Neumora Therapeutics Reports Data from KOASTAL-1 Study of Navacaprant in Major Depressive Disorder | Neumora Therapeutics, Inc. Study did not demonstrate statistically significant improvement on primary endpoint of reduction in depressive symptoms as measured by MADRS total score compared to placebo Navacaprant showed an efficacy signal in female participants; Company plans to further analyze results Navacaprant generally

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...

4DMT Announces Landmark Publication of 4D-150 Preclinical Data for the Treatment of Neovascular Retinopathies in IOVS Landmark publication in leading ophthalmology research journal demonstrates the power of 4DMT’s Therapeutic Vector Evolution platform to invent potentially best-in-class customized vectors and transfo...

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-...

Cidara Therapeutics Completes Enrollment of Phase 2b NAVIGATE Trial Evaluating CD388 for Prevention of Seasonal Influenza SAN DIEGO, Dec. 04, 2024 (GLOBE NEWSWIRE) -- Cidara Therapeutics, Inc. (Nasdaq: CDTX), a biotechnology company using its proprietary Cloudbreak® platform to develop drug-Fc conjugate (DFC) immunothera...

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-...

Review | ‘Gabriel’s Moon,’ by William Boyd, will keep readers guessing Boyd’s 18th novel is a spy thriller about a travel writer who gets recruited by MI6.

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

Not actually a word, I feel...

It probably will be soon!

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...

London Healthcare Week: Forbion Managing Partner Sander Slootweg shares his thoughts on the year ahead, the IPO market, M&A environment, areas of interest, and more Fresh off of raising more than €2 billion for two new funds in October, he gives his take on the biotech markets both in Europe and globally.Forbion Full

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

My 1st co Neurovex, spun out of UCL in 1999, started out trying for GT for neuro diseases: 3 yrs in we decided that trying to kill things (ie tumors) was going to be easier than trying fix things (ie GT), & hence Neurovex became Biovex - in reality, not a lot's changed re delivery between then & now

Which all in all makes it very hard for companies to successfully commercialize..

Gene therapy pioneer Jim Wilson believes he can commercialize rare-disease drugs in a tough climate Jim Wilson said he's leaving the University of Pennsylvania to show biotechs can successfully commercialize gene therapies for rare diseases.

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...

Done! #BioSky