Solid Biosciences Inc.

Solid Biosciences develops gene therapy candidates targeting neuromuscular and cardiac diseases. The company's pipeline centers on rare genetic disorders, with SGT-003 in phase 1/2 development for Duchenne muscular dystrophy and SGT-212 in phase 1b for Friedreich's ataxia. Additional candidates address cardiac conditions including catecholaminergic polymorphic ventricular tachycardia, dilated cardiomyopathy variants, and rare inherited diseases associated with specific genetic mutations. Most programs remain in preclinical or early-stage development.

The company operates a platform technology approach focused on adeno-associated virus (AAV) vector optimization. Core capabilities include capsid libraries, genetic regulatory elements such as promoters and UTRs, immunomodulation technologies, and dual gene expression technology designed to package multiple transgenes into single capsids. These platform components support both internal pipeline development and potential partnerships.

Solid Biosciences maintains collaborations with Ultragenyx Pharmaceutical and licensing agreements with academic institutions including the University of Washington, University of Missouri, and University of Florida, as well as the Italian research hospital ICS Maugeri. The company operates with approximately 100 full-time employees and is headquartered in Charlestown, Massachusetts.