United States – October 27, 2026 -- Fifteen people with Duchenne muscular dystrophy had received the investigational gene therapy SGT-003 in the INSPIRE DUCHENNE clinical study as of August 12, 2025, according to developer Solid Biosciences.
Ten trial sites span four countries
The multi-center, open-label trial now runs 10 active clinical sites in the United States, Canada, Italy, and the United Kingdom. More sites are expected to open later this year.
Solid Biosciences expects to have treated at least 20 patients aged 0 to under 12 by the end of 2025. It anticipates widening enrollment to older patients in 2026.
No treatment-related serious adverse events reported
The company says SGT-003 has been well tolerated so far, with no serious adverse events related to treatment. One participant had a mild rise in liver enzymes after dosing, during the steroid taper period. That patient has shown no symptoms, has had no changes to daily activities, and is doing well on steroids alone.
FDA meeting and a placebo-controlled trial are planned for Q4 2025
In the fourth quarter of 2025, Solid Biosciences expects to meet with the U.S. Food and Drug Administration to discuss regulatory pathways for SGT-003.
The company also intends to start a separate randomized, placebo-controlled trial outside the United States in the same quarter. The trial is designed to support potential global regulatory authorizations.
The study is registered on ClinicalTrials.gov as NCT06138639.