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Dyne Therapeutics Highlights New Preclinical Data For Duchenne Muscular Dystrophy Program

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  • Dyne Therapeutics Inc (NASDAQ: 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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