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Design Therapeutics Announces Start of Friedreich Ataxia Patient Dosing Ex-U.S. in its RESTORE-FA Phase 1/2 Multiple-Ascending Dose Trial of DT-216P2

Design Therapeutics (DSGN)Design TherapeuticsClinical readout

Initial Data from the Ongoing, Blinded Phase 1 Single-Ascending Dose Trial Has Demonstrated Favorable Safety and Pharmacokinetics IND Application Submitted to Expand RESTORE-FA Trial to U.S. Sites; Notice Received from FDA Placing Clinical Hold on IND Application to Open U.S.

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Design Therapeutics

GeneTAC Platform Design Therapeutics develops GeneTAC small molecules that bind genomic repeat sequences and modulate transcription. The platform is designed either to increase expression of a deficient gene or reduce expression of a toxic mutant gene. DT-216P2 DT-216P2 is being developed for Friedreich ataxia and is designed to increase...

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