DYN News & Events

"Dyne is entering a critical period of transformation as we advance programs from our clinically validated FORCE platform toward commercialization, with our first potential launch in DMD in less than 12 months, if Priority Review is granted and approval is received. The positive topline results we reported in December for z-rostudirsen further demonstrate the potential of the FORCE platform to safely and effectively deliver drug payloads broadly and deeply into targeted tissues, all with the goal of providing functional improvement to patients in urgent need of new therapies," said John Cox, president and chief executive officer of Dyne.

Dyne Therapeutics announced that the Ministry of Health, Labour and Welfare, MHLW, in Japan has granted Orphan Drug designation, ODD, for zeleciment basivarsen, for the treatment of myotonic muscular dystrophy type 1, DM1. Z-basivarsen is currently being evaluated in the Phase 1/2 ACHIEVE clinical trial in individuals with DM1. "By targeting the underlying biology of DM1, z-basivarsen has shown early and sustained improvements in myotonia, muscle strength and function, with a favorable safety profile," said Doug Kerr, M.D., Ph.D., chief medical officer of Dyne. "This designation in Japan, alongside those already granted in the U.S. and Europe, emphasizes the urgent need for new therapies and highlights the potential of z-basivarsen to deliver meaningful functional improvement for people living with DM1."