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.
Dyne’s PMO is currently in pre-clinical development with a target of 2022 for submission of an IND to enable clinical trials.
This program is sponsored by Dyne Therapeutics.
Dyne Therapeutics Presents at the PPMD 2021 Virtual Annual ConferencePre-recorded content for PPMD's 2021 Virtual Annual Conference On-Demand Library
Webinar: Transforming the Treatment Paradigm with Dyne Therapeutics
Dyne Therapeutics joined Parent Project Muscular Dystrophy (PPMD) for a webinar on March 31, 2021 to provide an overview of the company’s mission, its novel FORCE platform designed to revolutionize drug delivery to muscle, and the company’s Duchenne drug development program.