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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.
Fennec Pharmaceuticals Inc is a commercial-stage biopharmaceutical company. Its product candidate is PEDMARK which is sodium thiosulfate in a novel formulation for the prevention of cisplatin-induced hearing loss, or ototoxicity in children. The company principally operates in the United States.