Gene therapy for Duchenne is centered on the goal of successfully introducing a smaller, but efficient version of dystrophin into the muscle cell. Fordadistrogene movaparvovec 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.

Status

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.

Sponsor

This program is sponsored by Pfizer Inc.

Related Studies

Media Library