P26 CONNECT-EDO51: Trial designs to support the development of PGN-EDO51 for Duchenne Muscular dystrophy amenable to exon 51 skipping

PepGen's Enhanced Delivery Oligonucleotide (EDO) cell-penetrating peptide technology is engineered to optimize tissue delivery and cellular uptake of therapeutic oligonucleotides. The EDO PGN-EDO51 is being evaluated for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Phase 1 single dose data in healthy volunteers demonstrated the highest levels of oligonucleotide delivery to biceps muscles at 15 mg/kg (up to 50 nM) and exon skipping (up to 2% skipping by ddPCR) that have been reported after a single dose in healthy volunteers.

Source: Neuromuscular Disorders - October 1, 2023 Category: Neurology Authors: J. Larkindale, S. Vacca, J. Cormier, J. Shoskes, J. Goyal, A. Holland, P. Lonkar, J. Foy, M. Mellion Source Type: research