Parent Project Muscular Dystrophy is excited to announce a $350,480 programmatic investment in Myosana Therapeutics, Inc. (Myosana) to support the company’s early-stage development of a non-viral gene therapy delivery platform aiming to slow skeletal muscle degeneration and heart failure in Duchenne.

Duchenne is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in 5,000 live male births. Duchenne is caused by a change in the DMD gene that codes for the dystrophin protein. Gene therapy holds the promise of providing benefit to patients with Duchenne by introducing replacement versions (truncated or full length) of the dystrophin producing gene into the muscle cell, where no dystrophin is produced.

Current gene therapy trials aim to deliver a micro-dystrophin transgene to cells in the body by using a viral vector known as adeno-associated virus (AAV). However, several challenges exist in utilizing AAV, including limited gene size capacity (only one-third of the dystrophin gene can be “packaged” into AAV), inability to currently re-dose due to an immune system response, and lack of targeting to specific tissues.

Myosana’s technology, created by Co-Founders Nick Whitehead and Stan Froehner, aims to address the problems posed by AAV administration through their development of a non-viral platform complex that targets genes of any size to skeletal and cardiac muscle. Additionally, non-viral platforms may circumvent some of the immune response and re-dosing challenges posed by AAV delivery.

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.

Eric Camino, PhD, PPMD’s Vice President of Research and Clinical Innovation, explains:

“With this programmatic investment in Myosana, PPMD continues our cutting-edge approach to accelerate treatments that have the potential to end Duchenne for every single person impacted by the disease. There is compelling preliminary evidence showing that Myosana’s non-viral gene delivery platform complex can deliver full-length dystrophin to muscle tissue. This investment from PPMD will enable the Myosana team to further advance the development of their platform complex in the hopes of improving the health and function of dystrophic muscle in all people living with Duchenne.”

Steve Runnels, Chief Executive Officer of Myosana, explains:

“We are extremely pleased to receive this investment from PPMD.  This is an important milestone for Myosana and will help accelerate our novel platform technology for non-viral, full-length dystrophin, gene delivery.”

Nick Whitehead, Chief Scientific Officer of Myosana, describes:

“Our task is to use full length dystrophin gene therapy to dramatically improve patients living with this genetic disorder. Our muscle targeted, non-viral gene delivery platform potentially overcomes many of the limitations of AAV viral vectors to deliver micro-dystrophin genes.”

In addition to significant pre-clinical and academic research funding, PPMD makes investments in early-stage biopharmaceutical companies to catalyze development of novel therapies to treat Duchenne and Becker. This type of early stage industry investment (often referred to as venture philanthropy) allows 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. Learn more about PPMD’s robust Research Strategy, funding initiatives and strategies for accelerating drug development.