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.
Every participant received SGT-003 alongside a steroid-only prophylactic immunomodulation regimen. Solid says this design aims to support tolerability while limiting the treatment burden on patients and families. Detailed safety data as of a May 29, 2026, cutoff will be presented at PPMD.
Kevin Flanigan presents the full program at 1:30 PM ET
Kevin Flanigan, MD, of Nationwide Children's Hospital, is the principal investigator for INSPIRE DUCHENNE. He will give an overview of the SGT-003 program at 1:30 PM ET during the session "Gene Therapy: Today & Tomorrow." Solid encourages attending families to watch.
Phase 3 IMPACT DUCHENNE is enrolling in two countries
IMPACT DUCHENNE is a randomized, placebo-controlled trial of SGT-003. It is currently enrolling at clinical sites in Australia and Canada. Sites in the United States, United Kingdom and Europe are planned in the coming months, subject to regulatory authorizations.
Solid designed INSPIRE DUCHENNE and IMPACT DUCHENNE as one integrated program. It is intended to support global regulatory authorizations, including potential accelerated approval in the United States, and to build a picture of safety, biomarker profile and clinical effect.
Discussions with the U.S. Food & Drug Administration are ongoing. The company says it will share an update as they progress.