Design Therapeutics, Inc. is a biotechnology company. It is engaged in the research and development of GeneTACT molecules, which are 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. Its Friedreich ataxia (FA) program is focused on the development of a disease-modifying treatment. Its DT-216 is developed to overcome the FXN transcription impairment that causes FA. DT-216 is in Phase I clinical trial. The Company is also engaged in developing DT-216 to enable higher exposure and chronic administration for treatment of FA, known as DT-216P2, which uses the same drug substance, DT-216. Its FECD program is focused on the development of a potentially disease-modifying medical treatment for FECD, DT-168, which is in Phase I clinical trial. Its HD program is focused on the development of a potentially disease-modifying treatment for HD.
