Develops gene therapies and treatments for Duchenne muscular dystrophy (DMD), focusing on genetic diseases.
Solid Biosciences Inc. is dedicated to advancing therapies for Duchenne muscular dystrophy (DMD) within the United States. The company's primary focus lies in its lead product candidate, SGT-001, currently undergoing Phase I/II clinical trials. SGT-001 aims to facilitate the expression of functional dystrophin protein in patients' muscles, addressing a critical need in the treatment landscape for DMD. Solid Biosciences is also developing SGT-003, the next-generation gene transfer candidate designed to further enhance therapeutic outcomes for individuals with Duchenne muscular dystrophy.
In addition to its innovative gene therapy candidates, Solid Biosciences is actively pursuing platform technologies such as dual gene expression, which enables the packaging of multiple transgenes into a single vector. The company also focuses on developing novel capsids, essential components for delivering genetic material effectively to targeted cells. These advancements underscore Solid Biosciences' commitment to leveraging cutting-edge science and technology to tackle the challenges posed by DMD and other genetic disorders.
Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences Inc. operates with a collaborative spirit, evident in its partnership and licensing agreement with Ultragenyx Pharmaceutical Inc. This collaboration aims to jointly develop and commercialize new gene therapies, further strengthening Solid Biosciences' position at the forefront of innovative treatments for Duchenne muscular dystrophy.