Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), today announced a $500,000 programmatic investment in Myosana Therapeutics, Inc. (Myosana) to support the company’s continuing development and translation of a non-viral gene therapy delivery platform aiming to slow skeletal muscle degeneration and heart failure in Duchenne.
Duchenne is the most common muscular dystrophy in children, affecting approximately one in 5,000 live male births. It is a progressive disorder that causes muscles to become weaker over time until it affects the whole body. Duchenne is caused by a change in the DMD gene that codes for the dystrophin protein.
While the field of gene therapy continues to advance and the promise of AAV-mediated delivery of micro-dystrophin transgenes progresses, the Duchenne community has recognized many challenges with these therapies, such as immune responses to viral capsid and barriers to redosing. PPMD previously announced a $350,480 programmatic investment in Myosana in August 2021, to support the development of its non-viral gene therapy candidate that could potentially deliver full-length dystrophin systemically.
Utilizing a non-viral platform to target the delivery of transgenes to muscle has the potential to increase the safety of genetic therapies and enable redosing, a critical challenge facing the field. Additionally, Myosana’s platform has the unique ability to deliver a full-length copy of the dystrophin gene rather than a shortened dystrophin transgene as is currently being utilized.
If successful, such technology holds the potential to slow skeletal muscle degeneration and heart failure in order to enhance and extend the lives of people with Duchenne, as well as other neuromuscular diseases.
PPMD’s investment of $500,000 is part of a larger round of seed funding totaling $5 million for the development of Myosana’s program that will enable a pathway for continued optimization and development of their candidate, as detailed in Myosana’s press release earlier today.
In addition to significant pre-clinical and academic research funding, PPMD makes investments in early-stage biopharmaceutical companies as part of their Venture Philanthropy program to catalyze the development of novel therapies to treat Duchenne and Becker. These investments allow companies to complete critical studies needed to advance investigational products to the clinic, while also providing the potential to create a financial return for PPMD that can then be reinvested into additional Duchenne and Becker research programs in academia or industry. All of PPMD’s research investments (both academic and industrial) undergo a rigorous scientific evaluation and are selected on the basis of potential benefit to the Duchenne community.
To learn more about PPMD’s robust Research Strategy, funding initiatives, and strategies for accelerating drug development, click here.
ABOUT PARENT PROJECT MUSCULAR DYSTROPHY
We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won five FDA approvals.
Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org