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. Wave’s WVE-N531 is an exon skipping therapy utilizing their PN backbone chemistry modifications for those amenable to exon 53 skipping.
A Phase I/II trial is actively recruiting in the Canada and the UK.
This program is sponsored by Wave Life Sciences.
|Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy|