Dyne Therapeutics Presents Preclinical Data from its Duchenne Muscular Dystrophy Programs Targeting Exons 51 and 53 at World Muscle Society 2022 Congress
October 11, 2022 at 16:10 PM EDT
- Poster Highlights In Vivo Data for DYNE-251, Now Being Evaluated in Phase 1/2 DELIVER Clinical Trial in Patients Amenable to Exon 51 Skipping -
WALTHAM, Mass., Oct. 11, 2022 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced the presentation of preclinical data from its Duchenne muscular dystrophy (DMD) programs for the treatment of individuals with DMD mutations amenable to skipping exons 51 or 53. The data are featured in a poster at the 27th International Hybrid Annual Congress of the World Muscle Society taking place in Halifax, Nova Scotia, Canada from October 11-15, 2022.
“We are excited to present data which highlight the potential of our FORCE™ platform in building a global DMD franchise of exon skipping therapies. Beyond DYNE-251, our product candidate targeting exon 51, we are leveraging the modularity of the FORCE platform to rapidly identify candidates for other exons, including 53, 45 and 44. The poster being presented at World Muscle features foundational preclinical data underpinning the clinical development program for DYNE-251 and the first in vitro data for a FORCE conjugate targeting exon 53,” said Oxana Beskrovnaya, Ph.D., chief scientific officer of Dyne. “We look forward to reporting clinical data from our Phase 1/2 DELIVER trial of DYNE-251 anticipated in the second half of 2023, as this is an important milestone in our efforts to develop new exon skipping therapies for people living with this devastating disease.”
DMD is a rare disease caused by mutations in the gene that encodes for dystrophin, a protein critical for the normal function of muscle cells. Dyne’s FORCE platform targets the transferrin receptor 1, which is highly expressed on the surface of muscle cells. In DMD, FORCE is designed to deliver a phosphorodiamidate morpholino oligomer (PMO) to muscle tissue to promote the skipping of specific DMD exons in the nucleus, allowing muscle cells to create a truncated, functional dystrophin protein and potentially stop or reverse disease progression.
The poster (P.131) entitled, “Building a FORCE™ platform-based DMD franchise for the treatment of individuals with mutations amenable to exon skipping” will be available in the Scientific Publications & Presentations section of Dyne’s website.
About DYNE-251 and the DELIVER Trial
DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1) which is highly expressed on muscle. It is designed to enable targeted muscle tissue delivery and promote exon skipping in the nucleus, allowing muscle cells to create a truncated, functional dystrophin protein, with the goal of stopping or reversing disease progression. In preclinical studies with Dyne’s FORCE™ platform, robust and durable exon skipping and dystrophin expression were observed in the mdx mouse model in skeletal and cardiac muscle as well as reduced muscle damage and increased muscle function. DYNE-251 demonstrated a favorable safety profile and achieved impressive exon skipping in non-human primates, especially in the heart and diaphragm, muscles in people living with DMD that weaken over time leading to mortality.
In addition to DYNE-251, Dyne is building a global DMD franchise with preclinical programs for patients with mutations amenable to skipping other exons, including 53, 45 and 44.
About Duchenne Muscular Dystrophy (DMD)
About Dyne Therapeutics