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 1 study of healthy volunteers has been completed.
This program is sponsored by PepGen.
PepGen Therapies at the PPMD 2021 Virtual Annual Conference
Pre-recorded content for PPMD's 2021 Virtual Annual Conference On-Demand Library