Gene therapy for Duchenne is centered on the goal of successfully introducing a smaller, but efficient version of dystrophin into the muscle cell. PF-06939926 is an investigational, recombinant adeno-associated virus, serotype 9 (AAV9) carrying a shortened version of the dystrophin gene (mini-dystrophin). Because the human dystrophin gene is too large to fit in the AAV9 capsid, a mini-dystrophin was developed that may help retain muscle function similar to that of a patient with a more mild disease, like Becker muscular dystrophy.
A Phase 3 study is currently recruiting globally; no sites have been opened in the U.S. and are not expected to open in the first half of 2021. Click here to learn more.
This program is sponsored by Pfizer Inc.
ACTIVE, NO LONGER RECRUITING
|A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy|
|A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy|
Pfizer Presents at the PPMD 2019 Annual Conference
Pfizer Presents at the PPMD 2018 Annual Conference