Duchenne Gene Therapy SGT-003 Shows Early Promise in Trial
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.