ARTICLE | Product Development

Will a gene therapy approval in DMD harm innovation?

A deep pipeline of gene therapy candidates follows Sarepta’s SRP-9001, meaning patients may face a tough choice

May 19, 2023 11:29 PM UTC

Next week could bring the first FDA approval of a gene therapy for Duchenne muscular dystrophy, which would mark a major milestone for patients and the industry. It would also set patients up with a difficult choice — whether to take this treatment, or wait for the wave of innovation that could yield better options, since taking both may not be an option.

With a handful of compounds in the clinic, and a growing pipeline of new strategies in preclinical development, the field is poised to benefit from the advances in biology that are offering hope in this complex disease.  Several of these were on display this week at the American Society of Gene and Cell Therapy conference (see Table)...