PPMD has provided $400,000 in funding to MyoGene through PPMD Venture Pathways, the organization’s venture-philanthropy initiative that provides industry funding to accelerate therapeutic development for Duchenne and Becker.
What is MyoDys?
MyoGene’s strategy, known as MyoDys 45-55, utilizes gene editing technology to edit an individual’s DMD gene. They target a large deletion of exons 45-55, encompassing a hotspot of disease-causing variants and creating an in-frame deletion that restores dystrophin production. MyoGene’s approach mimics a deletion found in mild Becker muscular dystrophy, associated with production of a highly functional dystrophin protein. This type of deletion and re-framing of the DMD gene would be amenable for approximately 50% of individuals with Duchenne. Unlike gene therapies that deliver a dystrophin transgene where durability is unknown, editing the DNA represents a permanent alteration.
PPMD’s support will enable MyoGene to conduct key studies to advance towards the clinic. MyoGene will generate data comparing the functionality of their corrected 45-55 deletion dystrophin protein to other dystrophins and advance work on characterization of safety of this approach.
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