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Design Therapeutics, Inc.

Pipeline4 assets

  • DT-216P2DT-216P2 / FXN GAA repeat expansion / frataxin expression / GeneTAC small molecule / Genetic disease / neurology
    Friedreich ataxiaPhase 1/2RESTORE-FA multiple-dose trial opened/ongoing; evaluates IV and subcutaneous administrationex-U.S.
  • DT-168DT-168 / TCF4 CTG repeat expansion / GeneTAC small molecule eye drop / Ophthalmology / genetic disease
    Fuchs endothelial corneal dystrophyPhase 2Biomarker trial opened/ongoing in FECD patients scheduled for corneal transplant surgery
  • DT-818DT-818 / DMPK CTG repeat expansion / GeneTAC small molecule / Neuromuscular / genetic disease
    Myotonic dystrophy type 1Phase 1 plannedPatient dosing in Phase 1 MAD trial anticipated in H1 2026Australia
  • Huntington's disease GeneTAC programHTT CAG repeat expansion / mutant huntingtin expression / GeneTAC small molecule / Neurology / genetic disease
    Huntington's diseasePreclinicalProgram disclosed; candidate not named

Latest news7 items

All news

Design Therapeutics Announces Initiation of Patient Dosing in Phase 1 Multiple Ascending Dose Trial of DT-818 for Myotonic Dystrophy Type-1

GeneTAC ® small molecule designed to selectively reduce transcription of the mutant DMPK allele and address the underlying cause of DM1 CARLSBAD, Calif., June 30, 2026 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for

Trial Start • Phase 1Trial initiation Tue, Jun 30, 2026, 8:00 AM EDT

Design Therapeutics Announces Four-Week IV Data from the RESTORE-FA Trial of DT-216P2 Demonstrating Clinical Improvements and Comprehensive Biomarker Activity in Friedreich Ataxia

DT-216P2 demonstrated dose-dependent improvement in multiple clinical measures and increases in endogenous frataxin mRNA and protein after four weeks of intravenous dosing DT-216P2 generally well-tolerated Data support advancement toward registrational development Management to host conference call

Interim Data • Phase 1/2Interim data release Mon, May 18, 2026, 7:00 AM EDT

Design Therapeutics to Host Investor Webcast to Review Data from RESTORE-FA Trial of DT-216P2 for Friedreich’s Ataxia on Monday, May 18, 2026

CARLSBAD, Calif., May 17, 2026 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, will announce data from the ongoing Phase 1/2 RESTORE-FA trial evaluating DT-216P2 in patients with

Results Call • Phase 1/2Results call Mon, May 18, 2026, 8:00 AM ET

Design Therapeutics to Participate in Upcoming Investor Conferences

CARLSBAD, Calif., Feb. 18, 2026 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that management will present at the following upcoming investor conferences:

Design Therapeutics, Inc. • Endpoint Arena