other material
confidence high
sentiment positive
materiality 0.60
PepGen reports preclinical data: PGN-EDODM1 corrects 99% of mis-splicing in DM1 mouse model
PepGen Inc.
- EDO platform delivers 25-fold higher oligonucleotide to myotube nuclei vs naked in vitro.
- 72% of skeletal muscle nuclei in NHPs positive for oligo after two 30 mg/kg doses.
- Single dose of PGN-EDODM1 reversed 76% myotonia and corrected 68% mis-splicing in DM1 mouse.
- Four doses reversed 99% myotonia and corrected 99% mis-splicing; well-tolerated.
- PGN-EDO51 safety in humans: transient, mild, reversible TEAEs at 5 and 10 mg/kg.