fordadistrogene movaparvovec

Fordadistrogene movaparvovec is an investigational gene therapy developed for the treatment of Duchenne muscular dystrophy (DMD). It uses a recombinant adeno-associated virus serotype 9 (AAV9) vector to deliver a shortened, functional version of the human dystrophin gene—known as mini-dystrophin—into muscle cells. This approach aims to restore dystrophin protein expression in patients with DMD, who typically lack functional dystrophin due to mutations in the DMD gene. The therapy was designed as a one-time intravenous infusion and intended to slow or halt muscle degeneration by enabling muscle tissue to produce mini-dystrophin protein. Fordadistrogene movaparvovec was originally developed by Bamboo Therapeutics and later acquired and further developed by Pfizer. Despite promising early results, its development has been discontinued following phase III clinical trials.

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fordadistrogene movaparvovec