ARTICLE | Editor's Commentary
Another Jesse Gelsinger moment for gene therapy?
FDA, industry and patient groups should come together to learn the lessons from tragic, avoidable deaths from Sarepta’s gene therapies
July 19, 2025 12:02 AM UTC
For parents of non-ambulatory Duchenne muscular dystrophy patients who have been treated with Sarepta’s Elevidys gene therapy, “having this in your house is a 24/7 panic attack.” That’s what a Duchenne muscular dystrophy patient advocate told BioCentury after news broke about the third death from a Sarepta gene therapy.
The advocate has personal experience with the disease. To avoid jeopardizing relationships with the DMD community, she and other advocates BioCentury spoke with do not want to be identified...