Solid Biosciences Inc

NASDAQ:SLDB

Solid Biosciences, Inc. engages in the development of treatments for patients with Duchenne muscular dystrophy (DMD). The company is headquartered in Cambridge, Massachusetts and currently employs 104 full-time employees. The company went IPO on 2018-01-26. 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. The company is developing SGT-001 for the treatment of Duchenne through a single intravenous administration.