Spending the weekend in San Francisco with my 19yo son, doing tourist things and visiting my niece. It’s a nice change from being here running from hotel to hotel in the rain and talking about biopharma all day during JPM.

RT @MSollender: $ABVX Abivax Announces Positive Phase 3 Results from Both ABTECT 8-Week Induction Trials Investigating Obefazimod, its Fi….

$SRPT Voluntarily pausing all shipments of DMD gene therapy Elevidys in the US because “ it became obvious that maintaining [a] productive working relationship required this temporary suspension while we address any questions that FDA may have …” .

The media is not equating Elevidys in DMD with SRP-9004 for LGMD as $SRPT suggests. We all realize these two gene therapies have differences, but the fact remains that they use the same AAV vector & that vector is what is linked to patient deaths. We are not confused about that.

$SRPT Statement: Shortly after 2:30 p.m. ET today, Sarepta received an informal request from the FDA to voluntarily halt shipment of ELEVIDYS. We first heard of this potential request earlier in the day through media reports.

$SRPT The FDA asked Sarepta to stop shipping Elevidys and the company refused. It’s hard to see what is gained by digging in their heals here. FDA also revoked the platform technology approval for the LGMD gene therapies.

Confirmed: FDA is investigating Elevidys and asking $SRPT to stop shipping the gene therapy.

$SRPT A breathtaking interpretation of materiality in relation to patients’ lives, physician decision making, investors’ need to know.

$SRPT CEO Doug Ingram says the company did not believe the death in the limb girdle gene therapy trial of STP-9004 was material to disclose in Sarepta’s prior call because it was not related to Elevidys, FDA was aware and hadn’t changed its position, trial has been shut down ….

33 states, D.C. and Puerto Rico will participate in the Cell and Gene Therapy Access Model for Medicaid coverage of sickle cell treatments.

RT @MSollender: $RARE Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A….

After @SarahKarlin & I had time to reflect on our conversations & observations at #BIO2025, we recorded a podcast. Hope you enjoy our chat about policy issues, deal making & finance, and the year ahead for biopharma. @PharmaScrip @PharmaPinkSheet @Citeline

RT @BillGates: Health aid works.

Flying from Boston to San Diego this morning. Looking forward to relaxing & reflecting over the next few days on the conversations I had & panels I attended at #BIO2025 & reporting back next week & beyond about the #biopharma finance & deal environment, AI & more.

And here’s @PharmaScrip and Pink Sheet’s free #BIO2025 day 3 coverage.

And here’s our free #BIO2025 day 2 notebook.

Scrip’s and the Pink Sheet’s daily #BIO2025 notebooks with quick takes from some of our interviews and comments from speakers at the meeting are free to read without a subscription. Here’s our day 1 coverage.

RT @SarahKarlin: Trump was never directly called out by name, but BIO leadership discussed the risks posed by his policies during the seco….

RT @PharmaScrip: BIO Notebook: Policy Problems, ACIP’s Future, BI Is Popular, Makary On FDA-Industry Ties — Click image below to read more!….

RT @dgingery: The @PharmaPinkSheet has boots on the ground @IAmBiotech international convention. Here's the day 1 highlights from @SarahKar….