Preclinical autoimmune disease biotech Design Therapeutics files for a $100 million IPO

March 5, 2021, Design Therapeutics is a preclinical-stage biopharmaceutical company pioneering novel small-molecule therapeutic candidates, called gene targeted chimeras (GeneTACs), that are designed to be disease-modifying and target the underlying cause of inherited nucleotide repeat expansion diseases. The GeneTACs are designed to selectively bind to genetic repeat sequences, modulate gene expression either by restoring or blocking transcription, and restore cellular health. As a platform, the company believes that GeneTACs have broad applicability across monogenic nucleotide repeat expansion diseases. Design plans to initiate clinical trials with its lead product candidate in Friedreich ataxia patients to evaluate its safety, pharmacokinetics, and effect on frataxin levels by the first half of 2022, subject to receiving regulatory clearance to proceed into clinical trials.