Robert Coffin
@robertcoffin.bsky.social
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Ex-academic (many years ago) & biotech entrepreneur, including at Biovex & $REPL inc
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?
6 months ago
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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
12 months ago
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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
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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...
https://finance.yahoo.com/news/4dmt-announces-positive-interim-data-130000938.html
12 months ago
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$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...
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Replimune Announces RP2 Development Program Advances with First Patients Enrolled in Metastatic Uveal Melanoma and Hepatocellular Carcinoma Clinical Trials | Replimune
WOBURN, Mass. , Jan. 08, 2025 (GLOBE NEWSWIRE) -- Replimune Group, Inc. (NASDAQ: REPL), a clinical stage biotechnology company pioneering the development of novel oncolytic immunotherapies, today anno...
https://ir.replimune.com/news-releases/news-release-details/replimune-announces-rp2-development-program-advances-first
about 1 year ago
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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...
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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
https://ir.neumoratx.com/news-releases/news-release-details/neumora-therapeutics-reports-data-koastal-1-study-navacaprant
about 1 year ago
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Up 55% since I posted this - jolly good!
add a skeleton here at some point
about 1 year ago
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How it was, and how it's going, just days apart (and a 50F temperature difference too)
about 1 year ago
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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...
about 1 year ago
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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...
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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...
https://finance.yahoo.com/news/4dmt-announces-landmark-publication-4d-130000217.html
about 1 year ago
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A nice patient story with $REPL's RP1 (in this case in angiosarcoma)
cancer.uiowa.edu/news/iowan-s...
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Iowan sees new life after innovative cancer treatment | Holden
By participating in cutting-edge research at University of Iowa Health Care, Linda Jacobs beat cancer. Now, she is relishing time with her grandchildren, exploring new hobbies, and training a puppy.
https://cancer.uiowa.edu/news/iowan-sees-new-life-after-innovative-cancer-treatment
about 1 year ago
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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-...
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CG Oncology Announces Bond-003 Topline Results - CG Oncology |
Groundbreaking Cretostimogene Grenadenorepvec Monotherapy Data Demonstrates Sustained, Durable Complete Responses in High-Risk BCG-Unresponsive Non-Muscle Invasive Bladder Cancer – 74.5% of patients a...
https://cgoncology.com/cg-oncology-announces-bond-003-topline-results/
about 1 year ago
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In an age if increasing vaccine skepticism, I like this approach from $CDTX - a once yearly prophylactic antiviral flu shot (not a vaccine, nor dependent on the immune system), which should work for all flu strains and work whatever the immune status of the patient
finance.yahoo.com/news/cidara-...
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Cidara Therapeutics Completes Enrollment of Phase 2b NAVIGATE Trial Evaluating CD388 for Prevention of Seasonal Influenza
SAN DIEGO, Dec. 04, 2024 (GLOBE NEWSWIRE) -- Cidara Therapeutics, Inc. (Nasdaq: CDTX), a biotechnology company using its proprietary Cloudbreak® platform to develop drug-Fc conjugate (DFC) immunothera...
https://finance.yahoo.com/news/cidara-therapeutics-completes-enrollment-phase-130000598.html
about 1 year ago
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I will certainly be buying this:
www.washingtonpost.com/books/2024/1...
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Review | ‘Gabriel’s Moon,’ by William Boyd, will keep readers guessing
Boyd’s 18th novel is a spy thriller about a travel writer who gets recruited by MI6.
https://www.washingtonpost.com/books/2024/12/01/gabriels-moon-william-boyd-novel-review/?utm_campaign=wp_main&utm_medium=social&utm_source=twitter
about 1 year ago
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Winter has arrived in VT..16F this morning too
about 1 year ago
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$REPL receives Breakthrough Therapy designation for RP1 for advanced melanoma after anti-PD1 & submits BLA to the FDA
ir.replimune.com/news-release...
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https://ir.replimune.com/news-releases/news-release-details/replimune-receives-breakthrough-therapy-designation-rp1-and
about 1 year ago
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Worth a listen - Sander is great!
www.biotechtv.com/post/forbion...
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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
https://www.biotechtv.com/post/forbion-sander-slootweg-november-20-2024
about 1 year ago
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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
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https://www.wsj.com/real-estate/this-once-hot-real-estate-type-is-now-being-offered-as-office-space-3fde02d4?mod=e2tw#biosky
over 1 year ago
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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...
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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.
https://www.statnews.com/2024/08/22/gene-therapy-researcher-jim-wilson-penn-gemma-biotherapeutics-franklin-biolabs/
over 1 year ago
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