PPMD and MyoGene Bio (MyoGene) are excited to announce that 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. This funding commitment aims to advance the development of MyoGene’s gene editing strategy and potentially leverage additional funding from other awards to support studies that pave the way to the clinic.
Duchenne, the most common genetic disorder diagnosed in childhood that affects approximately one in 5,000 live male births, is caused by a change in the DMD gene that codes for the dystrophin protein. Gene editing is a strategy to make permanent alterations to an individual’s DNA to restore function of the gene.
MyoGene’s strategy, known as MyoDys45-55, 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 has been committed through our Gene Therapy Initiative to continue to push the field of gene therapy forward. Through PPMD Venture Pathways, we can help usher in yet another approach for treating patients with Duchenne,” explains Eric Camino, PhD, PPMD’s Vice President of Research and Clinical Innovation. “Gene editing represents a strategy to make life-long corrections to variants in patients’ DNA. Supporting the development of this approach will also further our understanding of novel precision medicine strategies to treat Duchenne.”
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.
PPMD’s investment and additional funds, including from Duchenne UK and How Women Invest, represent $1.42 million in total support for MyoGene. This funding commitment may be leveraged for additional non-dilutive grant funding, building on MyoGene’s previous success obtaining grants.
“Support from patient advocacy organizations like PPMD is vital to our work,” shares Courtney Young, PhD, CEO and Co-founder of MyoGene Bio. “This support not only provides critical funding to advance our research, but also sends a powerful signal to public and private funders about the importance of continued innovation in therapeutic development for Duchenne. With PPMD’s investment, we will be able to leverage patient-advocacy funding into larger awards, making this funding reach further as we advance development of our novel gene editing therapy for Duchenne.”
PPMD’s investment in MyoGene builds on the organization’s 30-year legacy of funding more than $55 million in Duchenne and Becker research to enhance our knowledge of dystrophinopathies. In addition to substantial preclinical and academic research funding, PPMD’s Venture Pathways program provides funding to biopharmaceutical companies, which allows companies to complete critical studies needed to advance investigational products to the clinic. These funding commitments also create the opportunity for PPMD to have a financial return that can be reinvested into its research strategy and support additional work in the future. All of PPMD’s research-funding commitments undergo rigorous scientific evaluation and are selected on the basis of potential benefit to the Duchenne and Becker community. Learn more about PPMD’s Venture Pathways program.
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