Company profile

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 news6 items

May 28, 2026

Design Therapeutics to Participate in 2026 Jefferies Global Healthcare Conference

CARLSBAD, Calif., May 28, 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 participate in a fireside chat during the 2026 Jefferies Global

May 18, 2026

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

May 17, 2026

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

Apr 28, 2026

Design Therapeutics Announces First Quarter 2026 Financial Results and Recent Business Updates

Additional Detail Provided for RESTORE-FA (DT-216P2) Trial Design, Dosing and Endpoints David Shapiro, M.D., Appointed to Board of Directors, Strengthening Clinical and Regulatory Expertise Cash and Securities of $222.8 Million at Quarter-End Provide Runway to Support Ongoing Clinical Execution

Mar 9, 2026

Design Therapeutics Reports Fourth Quarter and Full Year 2025 Financial Results and Recent Business Updates

Trials for DT-216P2 (RESTORE-FA) and DT-168 (FECD) Ongoing; DT-818 (DM1) Dosing in Patients Expected in the First Half of 2026 Cash and Securities of $219.8 Million as of Year-End Supports Ongoing Clinical Execution CARLSBAD, Calif., March 09, 2026 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc.

Feb 18, 2026

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: