SGT-003, an investigational microdystrophin gene therapy for Duchenne, continues to demonstrate an encouraging safety profile and has been generally well tolerated in 40 participants treated to date. Interim data show robust microdystrophin expression, restoration of key components of the dystrophin-associated protein complex (DAPC), improvements in biomarkers of muscle integrity, and stabilization or improvement in cardiac function following treatment. The company plans additional meetings with the FDA in the first half of 2026 to discuss a potential accelerated approval pathway and expects to provide a regulatory update mid-2026.
Read press release: https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-provides-interim-positive-clinical-update
Read community letter: https://www.solidbio.com/letter-to-the-duchenne-community-march2026/
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