Duchenne Gene Therapy SGT-003 Has Now Dosed 53 Boys in Trial
Global – October 27, 2026 -- Fifty-three boys with Duchenne muscular dystrophy have now received the investigational gene therapy SGT-003, and a Phase 3 trial is already enrolling patients in Australia and Canada. Solid Biosciences shared the update in a letter to the Duchenne community as the annual Parent Project Muscular Dystrophy (PPMD) conference got under way.
The Phase 1/2 trial spans ages 0 to 10
The count comes from the Phase 1/2 INSPIRE DUCHENNE trial and reflects a June 22, 2026, enrollment cutoff. Participants range from 0 to 10 years old. One of them is a 6-month-old boy who, to the company's knowledge, is the youngest child ever dosed with a microdystrophin gene therapy.
Solid reports no cases of four serious safety conditions
The company describes SGT-003 as generally well tolerated. It reports no observed cases of drug-induced liver injury, myocarditis, thrombotic microangiopathy or atypical hemolytic uremic syndrome.