delandistrogene moxeparvovec

Delandistrogene moxeparvovec is a recombinant adeno-associated virus (AAV) vector-based gene therapy designed to treat Duchenne muscular dystrophy (DMD). It utilizes the rAAVrh74 serotype to deliver a transgene encoding a shortened, functional version of the dystrophin protein, known as micro-dystrophin, under the control of the muscle-specific MHCK7 promoter. This micro-dystrophin retains critical functional domains, including the actin-binding and cysteine-rich domains, to stabilize the sarcolemma and protect muscle fibers from contraction-induced injury. Developed by Sarepta Therapeutics in collaboration with Roche and Nationwide Children's Hospital, the therapy received accelerated FDA approval in 2023 for ambulatory pediatric patients and was subsequently expanded to a full approval for ambulatory patients and accelerated approval for non-ambulatory patients aged 4 years and older.

Brand names

Elevidys

Other names

delandistrogene moxeparvovec-roklmicro-dystrophin gene therapy