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Industry Partner Update: Dyne Therapeutics

Starts July 8, 2024
11:30 AM
12:30 PM
Event Type: Virtual
Event Category: Research
Audience Type: Everyone
Dr. Ash Dugar Headshot

Defeat Duchenne Canada is committed to bringing the latest in Duchenne muscular dystrophy research and drug development to you – our Canadian Duchenne community.


Webinar Series: Advancing DYNE-251: An Investigational Medicine for DMD to Deliver for Patients

Dyne Therapeutics will present clinical data from the DELIVER Trial of DYNE-251 in individuals living with Duchenne muscular dystrophy who are amenable to exon 51 skipping, first released on May 20th, 2024.  These data demonstrated compelling impact on key disease biomarkers and trends in improvement of multiple functional endpoints.

Presented by: Ash Dugar, PhD, MBA- Chief Medical Affairs Officer

 

About DYNE-251

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.

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