Solid Biosciences Inc. engages in developing therapies for duchenne muscular dystrophy in the United States. The company's lead product candidate is SGT-001, a gene transfer candidate, which is in a Phase I/II clinical trial to drive functional dystrophin protein expression in patients' muscles; and SGT-003, a ext-generation gene transfer candidate for the treatment of duchenne muscular dystrophy. It also engages in developing of platform technologies, including dual gene expression, a technology for packaging multiple transgenes into one vector, as well as novel capsids. The company has collaboration and license agreement with Ultragenyx Pharmaceutical Inc. to develop and commercialize new gene therapies for Duchenne Muscular Dystrophy. Solid Biosciences Inc. was incorporated in 2013 and is headquartered in Cambridge, Massachusetts.
Page last updated on:
January 14th, 2023