by: Parent Project Muscular Dystrophy
Dyne Therapeutics recently joined PPMD for a community webinar to present initial clinical data from the DELIVER trial of DYNE-251, a next-generation exon skipping investigational product, in individuals with Duchenne who are amenable to exon 51 skipping.
The Dyne team highlighted that the safety profile for DYNE-251 is favorable, and pointed to the company’s proprietary FORCE™ platform as contributing to these positive results. The FORCE™ platform harnesses cell biology to deliver therapeutics such as DYNE-251 to muscle cells, which has the potential to reduce dosing frequency and chronic therapeutic administration. Initial data from DELIVER also showed that DYNE-251, when dosed monthly, resulted in an increase in percent dystrophin and percent dystrophin positive fibers at six months.
Dyne indicated that next steps for DYNE-251 include optimizing the dose and dose regimen, and expects the next clinical update from DELIVER in the second half of 2024. Finally, the Dyne team answered questions from the community on a variety of topics.
