PGN-EDO44

PGN-EDO44 is a peptide-conjugated antisense oligonucleotide (ASO) developed by PepGen for the treatment of Duchenne muscular dystrophy (DMD) in patients with mutations amenable to exon 44 skipping. It utilizes PepGen’s Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve cellular uptake and nuclear delivery of therapeutic oligonucleotides. In preclinical studies, PGN-EDO44 demonstrated high, dose-dependent levels of exon 44 skipping in wild-type human myoblasts—achieving up to 93.4% mean exon skipping at the highest dose tested. This mechanism aims to restore the open reading frame of the dystrophin gene transcript, enabling production of a truncated but functional dystrophin protein in DMD patients whose mutations are suitable for this approach[2][4][6][10]. Despite promising preclinical data, development has been discontinued and it did not advance beyond discovery/preclinical stages[1].