Solid Biosciences Inc.

Solid Biosciences Inc. is a precision genetic medicine company dedicated to advancing a portfolio of gene therapy candidates for patients suffering from rare neuromuscular and cardiac diseases. The company's mission is to improve the daily lives of individuals living with devastating rare diseases, particularly those directly impacted by Duchenne muscular dystrophy. Headquartered in Charlestown, MA, USA, Solid Biosciences focuses on developing transformative treatments through scientific innovation.

The company's pipeline includes several gene therapy candidates such as SGT-003 for Duchenne muscular dystrophy, SGT-212 for Friedreich's ataxia, SGT-501 for catecholaminergic polymorphic ventricular tachycardia, SGT-601 for TNNT2-mediated dilated cardiomyopathy, SGT-401 for BAG3-mediated dilated cardiomyopathy, and SGT-701 for RBM20-mediated dilated cardiomyopathy. Beyond specific product candidates, Solid Biosciences is also engaged in developing innovative platform technologies, including novel capsid libraries and genetic regulators, to enhance gene therapy delivery across various applications.

Led by President and CEO Bo Cumbo, Solid Biosciences has recently achieved significant milestones, including receiving European Commission Orphan Drug Designation for SGT-003 for Duchenne muscular dystrophy in April 2026. The company reported positive interim clinical data from its Phase 1/2 INSPIRE DUCHENNE trial for SGT-003 in March 2026 and announced an oversubscribed $240 million private placement. Solid Biosciences has also initiated a Phase 3 trial (IMPACT DUCHENNE) for SGT-003 and a Phase 1b trial (FALCON) for SGT-212 for Friedreich's ataxia in Q1 2026, further solidifying its position in the competitive gene therapy landscape.