Design Therapeutics Advances GeneTAC® Pipeline And Appoints Biotech Veteran To Board

Design Therapeutics, Inc.,  a clinical-stage biotech company focused on developing GeneTAC® small molecules for serious genetic disorders, announced key updates across its therapeutic pipeline alongside its Q3 2025 financial results. The company’s progress spans programs targeting Myotonic Dystrophy Type 1 (DM1), Friedreich Ataxia (FA), Fuchs Endothelial Corneal Dystrophy (FECD), and Huntington’s disease, reflecting continued momentum in advancing its portfolio of precision genetic therapies.

“The third quarter was marked by strong operational execution across our portfolio,” said Pratik Shah, Ph.D., chairperson and chief executive officer of Design Therapeutics. “Today we are excited to unveil DT-818 as our development candidate for the treatment of DM1, a debilitating neuromuscular disease with significant unmet medical need. With broad tissue distribution, significant splicing correction, and selectivity for mutant DMPK, we believe DT-818 has best-in-disease potential. Our DT-216P2 and DT-168 trials also continue to progress toward anticipated second half 2026 data readouts in FA and FECD. With these milestones, we are entering an exciting phase for Design as we advance multiple programs toward clinical proof-of-concept.”

For DM1, Design introduced DT-818 as its new development candidate. This GeneTAC® molecule targets the CTG repeat expansion in the DMPK gene, the root cause of DM1, by selectively reducing transcription of the mutant allele. Preclinical studies have shown DT-818 achieved more than a 90% reduction in toxic RNA foci and restored normal splicing in DM1 patient cells. With ex-US regulatory clearance obtained, the company plans to begin a Phase 1 multiple-ascending dose trial in Australia in early 2026, with initial splicing correction data expected in 2027.

Design also continues to advance its broader pipeline. The RESTORE-FA Phase 1/2 trial for DT-216P2 in Friedreich Ataxia is ongoing, with frataxin (FXN) expression data anticipated in late 2026. Similarly, its Phase 2 study of DT-168 in FECD patients undergoing corneal transplant will evaluate safety and biomarker responses, with results also expected in the second half of 2026. Additionally, preclinical work for a Huntington’s disease program is underway. In a governance update, Design appointed veteran biotech leader Justin Gover—former CEO of GW Pharmaceuticals—to its Board of Directors, bringing over 25 years of strategic and operational expertise.