eP213: Phase 2 multiple ascending-dose study of SRP-5051 PPMO in patients with DMD amenable to exon 51 skipping: Part A results

Peptide-conjugated phosphorodiamidate morpholino oligomers (PPMOs) are a next-generation chemistry platform in which a cell-penetrating peptide is conjugated to the phosphorodiamidate morpholino oligomer (PMO) backbone, with the goal of increasing tissue penetration, exon skipping, and dystrophin production with less frequent dosing. SRP-5051 is an investigational PPMO designed to skip DMD exon 51. Here we report results from the Part A dose-finding phase of MOMENTUM (NCT04004065), an ongoing phase 2 study of SRP-5051.