One of the most common types of mutations in the dystrophin gene occurs when a piece of the code in the middle of the gene is missing or deleted. By skipping additional segments of the dystrophin code called exons, the deletion can shift from an out-of-frame deletion to an in-frame deletion. Typically, an in-frame deletion results in a smaller, but still functional, dystrophin protein. This shortened protein is expected to act in a similar way to normal dystrophin, and so relieve some symptoms of Duchenne and hopefully result in a milder presentation. PepGen’s EDO51 is an exon skipping therapy bound to a peptide to help the therapy reach the muscle cells. PGN-EDO51 targets those amenable to exon 51 skipping.
A Phase 2 study is actively recruiting in Canada.
This program is sponsored by PepGen.
ACTIVELY RECRUITING | A Study Of PGN-EDO51 In Participants With Duchene Muscular Dystrophy Amenable To Exon 51-Skipping Treatment (CONNECT1-EDO51) |
JUNE 2021 | PepGen Therapies at the PPMD 2021 Virtual Annual ConferencePre-recorded content for PPMD's 2021 Virtual Annual Conference On-Demand Library |
