Design Therapeutics, Inc. (DSGN)

12/3/21. 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, of which there are more than 40 debilitating degenerative diseases. 

 In preclinical studies for our lead program, we have observed restoration of frataxin (FXN) levels in cells from Friedreich ataxia (FA) patients using our FA GeneTACs. 

 We plan to initiate clinical trials with our lead product candidate in FA patients to evaluate its safety, pharmacokinetics (PK) and effect on FXN levels by the first half of 2022, subject to receiving regulatory clearance to proceed into clinical trials.