PepGen

PepGen is a clinical-stage biotechnology company focused on developing oligonucleotide therapies for severe neuromuscular and neurological diseases. The company's proprietary Enhanced Delivery Oligonucleotide (EDO) platform leverages cell-penetrating peptides to improve the uptake and activity of conjugated oligonucleotide therapeutics. PepGen's lead candidate, PGN-EDO51, is being developed for Duchenne muscular dystrophy (DMD) and targets exon 51 skipping. In preclinical studies, PGN-EDO51 demonstrated exon 51 skipping levels of 58% in the tibialis anterior and 19% in the heart with a single intravenous dose of 20 mg/kg in non-human primates. The company is also advancing PGN-EDODM1 for myotonic dystrophy type one (DM1). In mouse models, a single dose of PGN-EDODM1 showed sustained correction of mRNA mis-splicing and complete amelioration of the DM1 myotonia phenotype for up to three months. PepGen's technology has shown the ability to deliver therapeutics to critical tissues, including skeletal, cardiac, and smooth muscle, as well as the central nervous system across the blood-brain barrier. The company plans to initiate clinical trials for PGN-EDO51 in early 2022 and accelerate the development of its DM1 program in late 2022. In October 2023, PepGen received clearance from the FDA to begin a Phase 1 study of its therapy for DM1 in the US, with proof-of-concept data expected in 2024.