Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disease caused by mutations in the dystrophin gene. This gene provides instructions for producing dystrophin protein, which stabilizes muscle cells. Without functional dystrophin, muscles weaken over time, leading to severe mobility loss, heart complications, and respiratory failure.
One promising therapeutic approach involves antisense oligonucleotides—molecules that help cells skip faulty sections of the dystrophin gene, restoring the production of a functional, though slightly shortened, protein. However, efficient delivery of these therapies to muscle cells has been a major challenge.
PepGen’s Enhanced Delivery Oligonucleotide (EDO) technology is designed to overcome this barrier. Their proprietary EDO peptides enhance the uptake of oligonucleotide therapies in skeletal, smooth, and cardiac muscle, significantly improving treatment effectiveness. Their leading candidate, PGN-EDO51, is designed to treat DMD patients amenable to exon 51 skipping, potentially benefiting around 13% of those living with the disease.
In preclinical and clinical studies, PGN-EDO51 has demonstrated unprecedented levels of exon skipping and dystrophin protein restoration. A Phase 1 trial in healthy volunteers showed high oligonucleotide delivery and strong exon skipping after a single dose, with good tolerability. If successful, this approach could slow or even halt disease progression, offering new hope to individuals affected by DMD.
Learn more about this technology at PepGen.com.
