United States – October 27, 2026 -- Families affected by Duchenne muscular dystrophy have early data on SGT-003, an investigational gene therapy from Solid Biosciences: 90 days after dosing, three boys showed average microdystrophin expression of 110%.
Three boys show muscle health improvements at 90 days
The Phase 1/2 INSPIRE DUCHENNE study reported initial data from its first three patients: two 5-year-old boys and one 7-year-old boy. Beyond microdystrophin expression, the company observed reductions in the muscle injury markers CK, AST, ALT, Titin, LDH and eMHC. It describes this as potential stabilization of muscle health.
The company also reports promising signs of muscle function preservation and encouraging early findings in potential cardiac benefit. Heart health is critically important in Duchenne. The company calls its outlook one of cautious optimism.
No serious adverse events across six dosed patients
SGT-003 has been well tolerated in the six patients dosed so far. The most common side effects were mild and typical of AAV-based gene therapies: nausea, vomiting, fever and temporary decreases in platelet levels. No serious adverse events were observed.
Enrollment targets about 20 patients by end of 2025
The study is actively enrolling at six clinical trial sites across the United States and Canada. It has regulatory approvals to expand to the U.K. and Italy. Solid Biosciences expects to enroll approximately 20 patients in total by the end of 2025.
In mid-2025, the company plans to request a meeting with the U.S. Food and Drug Administration to discuss a possible accelerated approval pathway for SGT-003.
Webinar and conferences give families a chance to ask questions
The company held a conference call on February 18 at 8:00 AM ET to discuss the findings. Parent Project Muscular Dystrophy will host a community webinar with Solid Biosciences team members on Tuesday, February 25 at 1:00 PM ET, with questions from the community welcome.
The team will also appear at two upcoming conferences: the Parent Project Italy 22nd Annual International Conference in Rome (February 28 to March 2, 2025) and the Duchenne UK New Horizons Conference in London (March 28 to 29, 2025).