Dyne Therapeutics Highlights New Preclinical Data For Duchenne Muscular Dystrophy Program

Dyne Therapeutics Inc DYN has announced new data presented today during the 2021 Muscle Study Group Annual Scientific Meeting for its Duchenne muscular dystrophy (DMD) program.
DMD is a rare disease caused by mutations in the gene that encodes for dystrophin, a protein critical for the normal function of muscle cells.
Dyne's FORCE platform targets the transferrin 1 receptor, which is highly expressed on the surface of muscle cells.
The level of dystrophin expression achieved with FORCE after a single dose in the mdx mouse model was substantial.
The dystrophin protein was widely expressed, with at least 80% dystrophin-positive fibers in skeletal and cardiac muscle.
Lead DMD program DYNE-251 achieved Exon 51 Skipping of 52% in the diaphragm and 43% in the heart in non-human primates.
It was well tolerated in GLP toxicology Study.
Related: Dyne Therapeutics' Myotonic Dystrophy Gene Therapy Shows Encouraging Action In Animal Studies.
Price Action: DYN stock is down 1.66% at $15.97 during the market session on the last check Friday.

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