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Solid Biosciences Inc is a life science company. It is engaged in the manufacturing of specialty and generic drugs. The company is involved in curing Duchenne muscular dystrophy (DMD), a genetic muscle-wasting disease predominantly affecting boys, with symptoms that usually manifest between three and five years of age. The company's pipeline products are the SB-001 and solid suit. The Company's singular focus is on developing treatments through gene therapy and other means for patients with neuromuscular and cardiac diseases. The company has categorized its programs into corrective therapies, disease-modifying therapies, and assistive devices.
Design Therapeutics Inc is a clinical-stage biopharmaceutical company engaged in the research and development of GeneTACTM molecules, which are a novel class of small-molecule gene-targeted chimera therapeutic candidates designed to be disease-modifying by addressing the underlying cause of diseases caused by inherited nucleotide repeat expansion mutations. The Company's product candidate is in Friedreich ataxia (FA), its second product candidate is in Fuchs endothelial corneal dystrophy (FECD), and it is also advancing its GeneTACTM program to address other serious nucleotide repeat-driven monogenic diseases.