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-212 for the treatment of Friedreich's ataxia, SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia, SGT-601 for the treatment of TNNT2-mediated dilated cardiomyopathy and additional assets for the treatment of genetic cardiac and neuromuscular disease. It also has two cardiac pipeline gene transfer programs, SGT-401 for BAG3-mediated DCM, which is in early preclinical development, and SGT-701 for RBM20 DCM, which is in the discovery stage. In addition to its gene transfer candidates, the Company has development programs focusing on platform technologies, including capsid libraries and dual gene expression, a technology that allows it to package multiple transgenes into one capsid.
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