DMD exon 53 skipping program / Dyne Therap - LARVOL DELTA

Building a FORCETM platform-based DMD franchise for the treatment of individuals with mutations amenable to exon skipping (WMS 2022) - "FORCE-M23D is a mouse-specific Fab-PMO conjugate designed to target TfR1 and skip exon 23 of the murine Dmd transcript to restore dystrophin expression in the mdx mouse model of DMD...Additionally, Dyne's clinical candidate, DYNE-251, a Fab-conjugated PMO designed to skip DMD exon 51, was evaluated in non-human primates (NHPs)...Lastly, we have begun development of FORCE conjugates for the treatment of exon 53 and exon 45 skipping amenable patients...This conjugate resulted in superior skipping of exon 53 compared to unconjugated PMO. Collectively, these data support utilizing the FORCE platform for the development of therapies for individuals with DMD mutations amenable to exon skipping."

Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • TFRC