Design Therapeutics, Inc. operates as a biotechnology company that develops therapies for serious degenerative disorders. The company is headquartered in Carlsbad, California and currently employs 58 full-time employees. The company went IPO on 2021-03-26. The firm is focused on gene-targeted chimeras (GeneTAC) molecules, which is a class of small-molecule gene-targeted chimera therapeutic candidates that are designed to be disease-modifying and target the underlying cause of inherited nucleotide repeat expansion diseases. The firm's lead product candidate is Friedreich ataxia (FA). The firm is also developing the GeneTAC program in myotonic dystrophy type-1 (DM1), and it is advancing its GeneTAC portfolio to address other serious nucleotides repeat-driven monogenic diseases. Its FA is a monogenic, autosomal recessive progressive disease. Its DM1 is a monogenic, autosomal dominant, progressive neuromuscular disease that affects skeletal muscle, heart, brain and other organs.

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