Design Therapeutics Reports RESTORE-FA Progress and Advances GeneTAC® Pipeline

CARLSBAD, Calif., Aug. 3, 2026

RESTORE-FA Trial Advances Following Positive Four-Week Data

Design Therapeutics is refining the ongoing RESTORE-FA clinical trial after encouraging four-week results reported in May 2026 demonstrated that DT-216P2 was generally well tolerated and produced dose-dependent increases in endogenous frataxin (FXN) mRNA and protein levels, along with improvements across multiple clinical measures in patients with Friedreich ataxia. The company plans to continue evaluating the 1 mg/kg dose as the intended go-forward regimen while expanding the 12-week cohort to approximately 10 patients. Additional protocol modifications include designating the percent change from baseline in endogenous blood FXN protein as the primary efficacy endpoint and exploring dose levels above 1 mg/kg. Design expects to provide an update on its registrational strategy during the fourth quarter of 2026, with 12-week clinical data anticipated in the first quarter of 2027.

GeneTAC® Pipeline Continues to Expand Across Genetic Diseases

Beyond Friedreich ataxia, Design Therapeutics continues advancing multiple GeneTAC® programs targeting serious genetic disorders. The company has initiated patient dosing in a Phase 1 multiple-ascending dose (MAD) trial evaluating DT-818 for myotonic dystrophy type 1 (DM1), with study data expected in 2027. In Fuchs endothelial corneal dystrophy (FECD), the ongoing Phase 2 biomarker trial of DT-168 continues evaluating safety, tolerability, and corneal biomarkers, although results are now expected in 2027 following manufacturing-related delays involving the drug delivery device. Design also continues advancing preclinical development of GeneTAC® candidates for Huntington’s disease, expanding the platform’s application across multiple inherited neurodegenerative disorders.

Strong Financial Position Supports Ongoing Clinical Development

Design Therapeutics reported research and development expenses of $16.4 million, general and administrative expenses of $5.8 million, and a net loss of $20.2 million for the second quarter ended June 30, 2026. The company ended the quarter with $207.4 million in cash, cash equivalents, and investment securities, providing financial resources to support its ongoing clinical programs and future development milestones. With continued progress across the RESTORE-FA program and multiple GeneTAC® candidates advancing through clinical and preclinical development, Design Therapeutics remains focused on developing targeted genomic medicines designed to precisely regulate disease-causing gene expression in severe genetic disorders.

Source: Design Therapeutics,press release