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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.
Price Action: DYN stock is down 1.66% at $15.97 during the market session on the last check Friday.
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