Solid Biosciences Inc. is a life sciences company that is focused on developing transformative treatments to improve the lives of patients with Duchenne muscular dystrophy (Duchenne). Duchenne is a genetic muscle-wasting disease predominantly affecting boys. SGT-001 and SGT-003 are its gene transfer candidates. SGT-001 and SGT-003 are designed to address the underlying genetic cause of Duchenne by delivering a synthetic transgene that produces dystrophin-like protein that is only expressed in muscles of the body, including cardiac and respiratory muscles. SGT-001 is its lead gene transfer candidate and utilizes an adeno-associated virus (AAV)9 vector. In addition to its gene transfer candidates, it has development programs focusing on platform technologies, including dual gene expression, a technology that allows it to package multiple transgenes into one vector, as well as capsids. It is developing SGT-001 for the treatment of Duchenne through a single intravenous administration.
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