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. Avidity’s AOC 1044 is an exon skipping therapy bound to a monoclonal antibody (mAb) to help the therapy reach the muscle cells. Avidity’s first AOC DMD is set to target those amenable to exon 44 skipping.

Status

AOC 1044 is currently in pre-clinical development with a target of 2022 for clinical trials.

Sponsor

This program is sponsored by Avidity Biosciences.

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