PepGen is committed to transforming clinical outcomes for individuals with Duchenne muscular dystrophy with our Enhanced Delivery Oligonucleotides (EDOs).
Oligonucleotides are genetic medicines that have the potential to modify the root cause of numerous neuromuscular disorders. Oligonucleotides for the treatment of DMD include exon skipping drugs. Some countries have approved exon skipping drugs for the treatment of DMD.
However, oligonucleotides, on their own, do not enter muscles easily, which limits their potential efficacy. For this reason, PepGen has developed peptides that can be conjugated (connected) to oligonucleotides, to enhance and improve their delivery and therefore their activity.
Preclinical research of our EDOs showed high levels of dystrophin production in animals.
Our phase 1 clinical study of males living without Duchenne suggests that EDOs deliver oligonucleotides to muscle, which supports clinical studies of EDO51 in males living with Duchenne.
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