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. Dyne’s FORCE platform is an exon skipping therapy bound to an antigen-binding fragment (Fab) to help the therapy reach the muscle cells. Dyne’s first product is set to target those amenable to exon 51 skipping.

Status

Dyne’s PMO is currently in pre-clinical development with a target of 2022 for submission of an IND to enable clinical trials.

Sponsor

This program is sponsored by Dyne Therapeutics.

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