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.
CIFFREO – A Phase 3 study is active, not recruiting; no sites have been opened in the U.S. Pfizer has reported the death of a patient in their non-ambulatory cohort of their Phase 1b study.
This program is sponsored by Pfizer Inc.
|A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy|
|CIFFREO - 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