Next-generation antisense therapies step forward with PepGen’s first Duchenne data

Targeted antisense therapy for DMD has cleared another hurdle, with the first look at data for PepGen’s PGN-ED051 suggesting the next-generation oligonucleotide conjugate is on the right track to provide a disease modifying effect.

PepGen Inc. (NASDAQ:PEPG) reported on Wednesday that PGN-ED051, the company’s lead cell-penetrating peptide-oligonucleotide conjugate based on its Enhanced Delivery Oligonucleotide (EDO) technology, led to mean exon 51 skipping levels of 2% following a single dose of 15 mg/kg in healthy volunteers in a Phase I study. The lower 10 mg/kg dose led to exon skipping of 1.4%. ...