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Solid Biosciences LLC

Solid Biosciences Inc. develops therapies for neuromuscular and cardiac diseases in the United States. Its lead product candidate, SGT-003, is in phase 1/2 for Duchenne muscular dystrophy. The company also develops SGT-212 for Friedreich's ataxia (phase 1b), SGT-501 for catecholaminergic polymorphic ventricular tachycardia (preclinical), SGT-601 for TNNT2-mediated dilated cardiomyopathy (preclinical), SGT-401 for BAG3-mediated dilated cardiomyopathy (preclinical), and SGT-701 for a rare inherited disease characterized by mutations in the RBM20 gene (discovery). It is also involved in developing platform technologies, including capsid libraries, genetic regulators such as promoters, UTRs, and introns, immunomodulation technologies, manufacturing purity, and dual gene expression. The company has a collaboration and license agreement with Ultragenyx Pharmaceutical Inc. and license agreements with the University of Washington, University of Missouri, University of Florida, and ICS Maugeri S.p.A. Solid Biosciences Inc. was incorporated in 2013 and is headquartered in Charlestown, Massachusetts.

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Biotech & Genomic Medicine

Solid Biosciences advances gene therapy pipeline with SGT-003 Phase 3 dosing and regulatory progress

Solid Biosciences has dosed the first patient in its Phase 3 IMPACT DUCHENNE trial of gene therapy SGT-003 for Duchenne muscular dystrophy, while continuing discussions with the FDA on a potential accelerated approval pathway. Interim Phase 1/2 results reported in March showed strong microdystrophin expression and improved muscle health in 40 treated participants, with an encouraging safety profile. The company also received a positive opinion from the European Medicines Agency on its Pediatric Investigation Plan for SGT-003 and was one of only three companies to receive the UK's Innovation Passport under the Innovative Licensing and Access Pathway. Solid's other clinical-stage gene therapies include SGT-212 for Friedreich's Ataxia, with initial Phase 1b data expected by year-end 2026, and SGT-501 for catecholaminergic polymorphic ventricular tachycardia, where the first participant is anticipated to be dosed in the second half of 2026. As of March 31, 2026, the company held $380.7 million in cash, cash equivalents, and available-for-sale securities, providing an operational runway into the first half of 2028.
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