Dyne Therapeutics presents new preclinical data on the potential of FORCE platform to address the genetic basis of Duchenne.
Dyne Therapeutics recently released new preclinical data from their Duchenne muscular dystrophy program, which was initially presented at the Muscle Study Group Annual Scientific Meeting. These data build upon previous data demonstrating the potential of the FORCE platform to address the genetic basis of Duchenne.
Dyne presented new mdx mouse data showing dystrophin restoration of 90% of wild-type levels in the diaphragm and 78% in the heart with 80% dystrophin-positive fibers after a single dose. It was also shown that DYNE-251, Dyne’s exon skipping candidate for those amenable to skipping exon 51, demonstrated impressive exon 51 skipping in non-human primates, including 52% in the diaphragm and 43% in the heart, and was well tolerated. There was evidence of durable exon skipping and dystrophin expression in both cardiac and skeletal muscles in in vivo models. PPMD is encouraged by the positive pre-clinical results and will continue to update the community as this product is translated to the clinic for evaluation of efficacy in human populations.
For the full release, click here.
PPMD has learned that Dyne will host an R&D Day on October 13, 2021 at 8 am EST in order to educate attendees about their programs and upcoming clinical trials in both Duchenne and Myotonic Dystrophy Type I . For more information on R&D day or to register, click here.