PGN-EDO53

PGN-EDO53 is a preclinical investigational oligonucleotide therapeutic developed by PepGen for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 53 skipping. It utilizes PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform, which leverages cell-penetrating peptides to improve the delivery and activity of conjugated oligonucleotides. The drug is designed to induce skipping of exon 53 in the dystrophin gene transcript, thereby restoring the open reading frame and enabling production of a functional, albeit shortened, dystrophin protein. In non-human primate studies, PGN-EDO53 demonstrated high levels of exon 53 skipping—up to nearly three times higher than comparator peptide-phosphorodiamidate morpholino oligonucleotide (PPMO) conjugates—suggesting potential for meaningful clinical benefit in DMD patients with relevant mutations[1][2]. As of mid-2024, PGN-EDO53 remains in preclinical development.