Posted by Michael Wonder on 02 Apr 2022
DT-216 granted FDA fast track designation for patients with Friedreich's ataxia
30 March 2022 - DT-216 is a novel GeneTAC gene targeted chimera small molecule designed to specifically target the GAA repeat expansion mutation, the underlying cause of FA, and restore frataxin gene expression.
Design Therapeutics today announced that it has completed dosing in the first single ascending dose cohort of its Phase 1 clinical trial of DT-216 in patients with Friedreich's ataxia.
