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

About this update from Design Therapeutics, Inc.

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 serious degenerative genetic diseases, today announced the initiation of patient dosing in its Phase 1 multiple-ascending dose (MAD) clinical trial evaluating 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 and address the underlying cause of DM1. "Initiation of patient dosing with DT-818 marks an important milestone for Design and for the DM1 community," said Pratik Shah, Ph.D., chairperson and chief executive officer of Design Therapeutics. "DT-818 is designed to address the underlying genetic cause of DM1 by selectively reducing transcription of the mutant DMPK allele that gives rise to toxic RNA foci and downstream spliceopathy. We believe DT-818 has the potential to be a best-in-disease treatment for people living with DM1, supported by its differentiated preclinical profile, and we look forward to advancing its clinical development." The Phase 1 trial is an open-label, MAD study designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamic effects of DT-818 in adults with DM1. Design anticipates reporting data from this study in 2027. About Myotonic Dystrophy Type-1 and DT-818DM1 is a monogenic, autosomal dominant, progressive neuromuscular disease that affects skeletal muscle, heart, brain and other organs. The cardinal features include muscle weakness, myotonia (slow muscle relaxation) and early cataracts. In addition, affected individuals often experience cardiac arrhythmias and changes in neuropsychological function. DM1 is caused by a mutation in the DMPK gene and is estimated to affect more than 70,000 people in the United States. DT-818 is a GeneTAC® small molecule designed to address the genetic cause of DM1 by preventing the expression of mutant gene product and consequently of pathogenic nuclear foci. In preclinical studies, DT-818 demonstrated broad tissue distribution, selective targeting of mutant DMPK and robust pharmacodynamic activity, supporting its potential as a best-in-disease treatment approach for people l...

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