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-251 is an exon skipping therapy bound to an antigen-binding fragment (Fab) to help the therapy reach the muscle cells for those amenable to exon 51 skipping.
A Phase 1/2 study is currently recruiting.
This program is sponsored by Dyne Therapeutics.
ACTIVELY RECRUITING | DELIVER - Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping |
APRIL 2023 | Webinar: Dyne Therapeutics — Advancing FORCE in the Pursuit of Therapies (April 2023)Dyne Therapeutics joined PPMD for a community webinar on April 12, 2023 to discuss Dyne’s FORCE™ platform. |
JULY 2022 | Webinar: Dyne Therapeutics Update on DYNE-251 (July 2022)Dyne Therapeutics joined PPMD for a community webinar on Wednesday, July 20, 2022. Dyne provided an update on the recent U.S. Food and Drug Administration (FDA) announcement lifting the DYNE-251 clinical hold and clearing the Investigational New Drug (IND) application. Dyne will initiate a clinical trial of DYNE-251 in individuals with Duchenne amenable to skipping exon 51 and expects to begin dosing trial participants in mid-2022. |
JUNE 2021 | Dyne Therapeutics Presents at the PPMD 2021 Virtual Annual ConferencePre-recorded content for PPMD's 2021 Virtual Annual Conference On-Demand Library |
MAR 2021 | Webinar: Transforming the Treatment Paradigm with Dyne TherapeuticsDyne 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. |
