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
A Phase 1/2 study is currently recruiting healthy volunteers.
Sponsor
This program is sponsored by Avidity Biosciences.
Related Studies
ACTIVELY RECRUITING | EXPLORE 44 - Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping |
Media
FEB 2023 | Webinar: Avidity Biosciences – EXPLORE44 Clinical Trial Investigating AOC 1044 (February 2023)Avidity Biosciences joined PPMD for a community webinar on February 8, 2023 to discuss the EXPLORE44™ clinical trial. |
JUNE 2021 | Avidity Biosciences at the PPMD 2021 Virtual Annual ConferencePre-recorded content for PPMD's 2021 Virtual Annual Conference On-Demand Library |
