Solid Biosciences, Inc., has shared updates on the company’s gene therapy program for the treatment of Duchenne. According to Solid, 33 participants have now been dosed in the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003. SGT-003 is a gene therapy candidate for the treatment of Duchenne that delivers a microdystrophin via their novel AAV capsid, which is designed to increase skeletal and cardiac muscle targeting while detargeting the liver.
SGT-003 continues to be well tolerated. According to Solid, the favorable safety and tolerability profile to date supports a steroid-only prophylactic immunomodulation regimen. Cardiac safety monitoring has shown reductions in cardiac injury and early signals of cardiac systolic function normalization, as measured by left ventricular ejection fraction (LVEF) in 14 individuals evaluated.
Additionally, Solid reports that in the first 10 participants evaluated, researchers observed strong levels of microdystrophin being produced in muscle cells. This was accompanied by restoration of important proteins that help stabilize and protect muscle fibers, along with improvements in multiple lab markers of muscle health.
Outside of the U.S., Solid reports that the first participant has been enrolled in IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled clinical trial, with dosing expected in Q1 2026.
According to Solid, the company plans to have multiple interactions with the U.S. Food and Drug Administration (FDA) during the first half of 2026 to align on its Phase 3 confirmatory trial design and on necessary confirmatory evidence required to support potential accelerated approval for SGT-003, with an update expected mid-year 2026. Solid expects to report additional data from the Phase 1/2 INSPIRE DUCHENNE trial in mid-2026.
Read Solid’s press release and community letter for more information.
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