Wednesday, November 10, 2021

Clinical Initiation of Lead GeneTACTM Program for Friedreich Ataxia On-track for the First Half of 2022

CARLSBAD, Calif., Nov. 09, 2021 (GLOBE NEWSWIRE); Design Therapeutics Reports Pipeline Progress and Third Quarter 2021 Results 

Friedreich Ataxia (FA) GeneTACTM Program On-track for Clinical Initiation in First Half of 2022: In ongoing IND-enabling studies, Design’s FA GeneTACTM clinical candidate has been shown to be well tolerated in repeat dose GLP toxicity studies in rats and non-human primates at doses that exceed what we estimate to be biologically active in the clinic. Design remains on track to initiate a Phase 1 clinical trial in patients with FA in the first half of 2022, with initial topline clinical data expected in 2022.