Key information

🔎 Fund Overview

Solid Biosciences Inc. is a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases. The Company is focused on advancing a portfolio of gene therapy candidates including SGT-003 for the treatment of Duchenne muscular dystrophy, SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia, AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, and additional assets for the treatment of fatal cardiac diseases. The Company also has two cardiac pipeline gene transfer programs, SGT-601 for TNNT2 DCM and SGT-701 for RBM20 DCM, that are both in early preclinical development. It also has a neuromuscular gene transfer program for the treatment of Friedreich's ataxia. In addition to its gene transfer candidates, the Company has development programs focusing on platform technologies, including novel capsid libraries and dual gene expression, a technology that allows it to package multiple transgenes into one capsid.