Design Gets FDA OK to Start Phase 1 Trial of DT-216 for FA

The U.S. Food and Drug Administration (FDA) has cleared Design Therapeutics to begin a Phase 1 clinical trial investigating its GeneTAC small molecule DT-216 for the treatment of Friedreich’s ataxia (FA). This clearance comes after the submission of an investigational new drug (IND) application based…

First Clinical Trial of GeneTAC Therapy for FA Expected in 2022

Design Therapeutics announced that it remains on track to launch the first clinical trial of its lead experimental candidate — gene-targeted chimera (GeneTAC) — for the treatment of Friedreich’s ataxia (FA) for the first half of 2022. The planned Phase 1 trial will test the therapy in patients,…