02:22 PM EDT, 03/11/2026 (MT Newswires) -- Solid Biosciences ( SLDB ) on Wednesday reported interim results from its phase 1/2 trial of SGT-003, a gene therapy being developed to treat Duchenne muscular dystrophy.
So far, 40 participants have received the treatment. The company said the therapy has been generally well tolerated and that interim analyses suggest robust microdystrophin expression, reaching up to 91% by day 360 in biopsies, along with evidence of improved muscle integrity and cardiac function.
The trial is testing a single intravenous dose of SGT-003 in children with Duchenne across 15 clinical sites in the US, Canada, Italy and the UK, and dosing is ongoing.
Solid said it plans to meet with the US Food and Drug Administration in H1 to discuss a potential accelerated approval pathway for SGT-003, with additional regulatory and clinical updates expected by midyear.
Shares of Solid Biosciences ( SLDB ) were down more than 12%.
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