Mandy Jackson

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.

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

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 insights.citeline.com/scrip/conferen…

Trump was never directly called out by name, but BIO leadership discussed the risks posed by his policies during the second day of the trade association’s annual convention..... Our #BIO2025 notebooks with quick roundups from the day are free to read. insights.citeline.com/pink-sheet/age…

The @PharmaPinkSheet has boots on the ground @IAmBiotech international convention. Here's the day 1 highlights from @SarahKarlin and @ScripMandy: BIO Notebook: PRVs Need To Be A Priority, IPO Window Shut And Focusing On Gene Therapy Safety insights.citeline.com//pink-sheet/co…

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.

$ABVX Abivax Announces Positive Phase 3 Results from Both ABTECT 8-Week Induction Trials Investigating Obefazimod, its First-in-Class Oral miR-124 Enhancer, in Moderate to Severely Active Ulcerative Colitis globenewswire.com/news-release/2…

$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 …” investorrelations.sarepta.com/news-releases/…

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

$RARE Ultragenyx Receives Complete Response Letter from FDA for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) globenewswire.com/news-release/2…

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.

BIO Notebook: Policy Problems, ACIP’s Future, BI Is Popular, Makary On FDA-Industry Ties — Click image below to read more! || #Scrip | Start your free trial today: vist.ly/3n769v4 vist.ly/3n769v5