PepGen is committed to transforming the treatment of people living with DMD 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.
In our phase 1 clinical study in healthy volunteers, we observed high levels of exon skipping in muscle.
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