DT-818

June 30, 2026: Design Therapeutics, Inc. (NASDAQ: DSGN) announced initiation of patient dosing in its Phase 1 multiple-ascending dose (MAD) clinical trial of DT-818 in adults with myotonic dystrophy type-1 (DM1). DT-818 is a GeneTAC small molecule designed to selectively reduce transcription of the mutant DMPK allele to address the underlying genetic cause of DM1.

The open-label Phase 1 MAD study will assess safety, tolerability, pharmacokinetics, and pharmacodynamic effects of DT-818 in DM1 patients. The company expects to report data from the study in 2027.

The release also notes DT-818’s preclinical profile, including broad tissue distribution, selective targeting of mutant DMPK, and robust pharmacodynamic activity. Design Therapeutics lists additional programs: DT-216P2 (Friedreich ataxia), DT-168 (Fuchs endothelial corneal dystrophy), and a Huntington’s disease program.