ELEVIDYS
ELEVIDYS (delandistrogene moxeparvovec) is a one-time gene therapy for Duchenne muscular dystrophy — a fatal progressive muscle-wasting disease caused by dystrophin gene mutations — developed by Sarepta Therapeutics. The therapy uses a hollow AAVrh74 virus to deliver a shortened but functional microdystrophin gene into muscle cells throughout the body, enabling patients to produce a form of the missing structural protein. ELEVIDYS received accelerated FDA approval in 2023 as the first gene therapy for DMD; Sarepta is pursuing a label expansion to ambulatory patients aged 4 and older.
Upcoming catalysts
Programs
Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a fatal X-linked muscle disease affecting roughly 1 in 3,500 boys that causes progressive muscle degeneration, loss of ambulation in early adolescence, and death from cardiac or respiratory failure before age 30; there is no cure and no prior gene therapy. ELEVIDYS was granted accelerated approval in 2023 for ambulatory patients aged 4–5; this supplemental program targets label expansion to all ambulatory DMD patients aged 4 and older, supported by functional outcome data from ongoing studies.