P25 Single- and repeat-dose nonclinical data for PGN-EDO51 demonstrate potential for the treatment of Duchenne muscular dystrophy (DMD)

PepGen's enhanced delivery oligonucleotide (EDO) cell-penetrating peptide technology is engineered to optimize tissue delivery and cellular uptake of therapeutic oligonucleotides. PGN-EDO51 is PepGen's clinical candidate for the treatment of people with DMD amenable to exon 51 skipping. In the mdx mouse model of DMD, a single intravenous (IV) dose of 30 or 60 mg/kg of mPGN EDO23 (mouse analogue of PGN-EDO51) resulted in exon skipping levels of 52.5% and 82.8% and dystrophin protein production of 22.5% and 51.7% in biceps.

Source: Neuromuscular Disorders - October 1, 2023 Category: Neurology Authors: A. Holland, P. Lonkar, C. Sweeney, J. Gilbert, N. Svenstrup, J. Goyal Source Type: research