United States – October 29, 2026 -- Solid Biosciences has dosed 33 children aged 1 to 10 with its investigational gene therapy SGT-003 for Duchenne muscular dystrophy, and a Phase 3 trial has enrolled its first patient.
Safety data show no liver, kidney or heart-inflammation cases
The figures come from INSPIRE DUCHENNE, a Phase 1/2, open-label, multi-center study, with data cut off on January 9, 2026. SGT-003 has been generally well tolerated using prophylactic steroids alone for immune suppression. The company reports no cases of drug-induced liver injury, thrombotic microangiopathy, atypical hemolytic uremic syndrome or myocarditis.
Cardiac monitoring has shown consistent reductions in markers of cardiac injury, with early signals of normalization. The company also reports microdystrophin expression and improvements across multiple biomarkers of muscle health.
Phase 3 trial will dose its first patients in Q1 2026
IMPACT DUCHENNE is a randomized, double-blind, placebo-controlled study of SGT-003's efficacy. Dosing is expected to begin in the first quarter of 2026. The trial runs outside the United States, with the first sites activated in Canada and Australia. Expansion to European countries is planned from mid-2026, subject to regulatory approvals.
The company is pursuing an integrated U.S. and ex-U.S. pivotal trial strategy to support potential accelerated regulatory authorizations.
Families can meet the team in London and Rome
Solid Biosciences plans to attend Duchenne UK's New Horizons Conference in London on February 6–7. It will also be at Parent Project's 23rd Annual International Duchenne and Becker Conference in Rome from February 27 to March 1. Trial details are listed on ClinicalTrials.gov under NCT06138639 (INSPIRE DUCHENNE) and NCT07160634 (IMPACT DUCHENNE).