Design Therapeutics Inc., a clinical-stage biotechnology firm focused on treating degenerative genetic diseases, announced the beginning of patient dosing in its RESTORE-FA Phase 1/2 multiple-ascending dose trial of DT-216P2 for Friedreich ataxia outside the United States. Initial data from a Phase 1 single-ascending dose trial in healthy volunteers indicated favorable safety and pharmacokinetics for DT-216P2. Design Therapeutics has submitted an investigational new drug $(IND.AU)$ application to the U.S. FDA to expand the trial to U.S. sites. However, the FDA issued a clinical hold on the IND application, citing nonclinical deficiencies. The company intends to address these issues promptly to continue their development efforts in the U.S. The trial is currently open for enrollment in Australia, with data anticipated in 2026.
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