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. Sites Forward-Looking Statements "We are pleased by the initial findings from the SAD trial that demonstrated a favorable safety profile for DT- 216P2 and support its advancement in the MAD FA patient trial. We are surprised by FDA's n ...

Phase 1 Single Ascending Dose Trial of DT-216P2 for Friedreich Ataxia (FA) Program Ongoing Well-Capitalized with Cash and Securities of $229.7 Million to Fund Operations Through up to Four Potential Clinical Proof-of-Concept Data Sets CARLSBAD, Calif., May 07, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced progress across its portfolio of GeneTAC candidates and reported ...