PEPG Profile
PepGen Inc. is a privately held, clinical-stage biotechnology company headquartered in Durham, North Carolina, USA. It was founded in 2019 with a focus on developing transformative therapies for people with neuromuscular diseases.
PepGen's platform technology involves the use of peptides to create new classes of drugs that are designed to target and treat genetic diseases caused by mutations in RNA splicing. The company's lead candidate, PGN-001, is a peptide-based therapy designed to restore dystrophin expression in patients with Duchenne muscular dystrophy (DMD).
PGN-001 uses PepGen's proprietary STAR technology, which stands for Structured Trans-splicing Activator of RNA. This technology is designed to induce RNA splicing in a way that restores the reading frame of the dystrophin gene, thereby allowing the production of functional dystrophin protein.
In addition to DMD, PepGen is exploring the potential of its platform technology for the treatment of other neuromuscular diseases caused by RNA splicing defects.
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