P27 Three novel enhanced delivery Oligonucleotide candidates for Duchenne muscular dystrophy mediate high levels of exon 53, 45, and 44 skipping

PepGen's enhanced delivery oligonucleotide (EDO) cell-penetrating peptide technology is engineered to optimize tissue delivery and cellular uptake of therapeutic oligonucleotides. PGN-EDO53, PGN-EDO45, and PGN-EDO44 are being evaluated for potential treatment of Duchenne muscular dystrophy (DMD) subpopulations amenable to exon 53, exon 45, or exon 44 skipping, respectively. Wild-type human myoblasts were dosed with PGN-EDO45 versus R6G-PMO45 (comparator PPMO), and with PGN-EDO44. High levels of exon skipping were seen for both EDO candidates.

Source: Neuromuscular Disorders - October 1, 2023 Category: Neurology Authors: A. Holland, P. Lonkar, C. Sweeney, H. Zhang, N. Svenstrup, C. Gibbons, L. Xu, J. Foy, J. Goyal Source Type: research