PepGen Presents Data from its Duchenne Muscular Dystrophy Program at World Muscle Society Congress

 Poster Details:

Title: Unlocking the potential of oligonucleotide therapeutics for Duchenne muscular dystrophy through enhanced delivery

• A single dose of PGN-EDO23 in the murine mdx DMD model achieved up to 93.1% exon skipping and 99.7% dystrophin expression in skeletal muscle, and 62.3% exon skipping and 25.7% dystrophin expression in the heart

• Three doses of the clinical product candidate, PGN-EDO51, in non-human primates achieved up to 78% exon skipping in skeletal muscle and 24% exon skipping in the left ventricle of the heart

• Repeat dosing of the clinical product candidate, PGN-EDO51, in non-human primates demonstrated accumulation of exon skipped transcript with each dose in quadriceps and biceps
  

https://finance.yahoo.com/news/pepgen-presents-data-duchenne-muscular-170000149.html