Key information

🔎 Fund Overview

Solid Biosciences Inc. is a life sciences company that is focused on developing transformative treatments to improve the lives of patients with neuromuscular and cardiac diseases. SGT-001 and SGT-003 are its gene transfer candidates. Its neuromuscular and cardiac programs, including SGT-003, a differentiated gene transfer candidate, for the treatment of Duchenne, AVB-202, a gene transfer candidate for the treatment of Friedreichs ataxia, and AVB-401 for BAG3 mediated dilated cardiomyopathy. Its 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. In addition to gene transfer candidates, the Company has development programs focusing on platform technologies, including novel capsid libraries and dual gene expression, a technology that allows to package multiple transgenes into one vector.