Myotonic Dystrophy Type 1

Overview

Autosomal dominant neuromuscular disease caused by CTG repeat expansion in the DMPK gene with nuclear RNA pathobiology; an entirely underserved indication targeted by Dyne Therapeutics' Dyne-101 using ASO-based DMPK knockdown. Myotonic dystrophy type 1 (DM1) has a nuclear RNA pathobiology caused by mutant DMPK messenger RNA, requiring an ASO delivered to the nucleus — which the FORCE platform uniquely achieves via systemic IV administration rather than intrathecal injection. DM1 is a totally underserved market with no approved disease-modifying therapy; Dyne Therapeutics' Dyne-101 is in a phase 3 registrational trial pursuing accelerated approval based on the DMPK biomarker. (Segment 5 (20:00-25:00))