Industry Partner Update: Dyne Therapeutics
Defeat Duchenne Canada is committed to bringing the latest in Duchenne muscular dystrophy research and drug development to you – our Canadian Duchenne community.
Join Ash Dugar, Ph.D., MBA, Dyne’s Chief Medical Affairs Officer, for an update on the initial clinical data, first released on January 3, 2024 (click the ‘press release’ button to the left), from the DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
DYNE-251 is Dyne’s product candidate being developed for people living with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. 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. In non-human primates, DYNE-251 demonstrated a favourable safety profile and achieved impressive exon skipping, 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 the DELIVER Trial
The DELIVER clinical trial of DYNE-251 is currently underway in Canada at the London Health Sciences Centre (London, Ontario) and the Children’s Hospital of Eastern Ontario – CHEO (Ottawa, Ontario). Recruitment is complete through the 20mg/kg cohort, but Dyne is continuing to progress the study with more cohorts up to 40mg/kg. If you are interested in participating in this clinical trial, we encourage you to speak to your healthcare team.