Design Therapeutics Announces FDA Clearance of Investigational New Drug Application for First GeneTACâ„¢ Molecule for Friedreich Ataxia

Design Therapeutics, Inc., a biotechnology company developing treatments for degenerative genetic disorders, announced that its Investigational New Drug Application (IND) for its lead candidate, DT-216, for the treatment of Friedreich ataxia (FA), was cleared by the U.S. Food and Drug Administration (FDA). The company is preparing to initiate a Phase 1 clinical trial of DT-216 to assess the safety, tolerability, pharmacokinetics, and frataxin (FXN) levels in patients with FA. Study enrollment is expected to begin in the coming weeks. DT-216 is a GeneTACâ„¢ gene targeted chimera small molecule designed to specifically target the GAA repeat expansion mutation and restore FXN gene expression.

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