Parent Project Muscular Dystrophy Invests $350,480 in Myosana Therapeutics to Support Non-Viral Gene Therapy Development

WASHINGTON, Aug. 26, 2021 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), today announced a $350,480 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.

"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," said Eric Camino, PhD, PPMD's Vice President of Research and Clinical Innovation. "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."

"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," said Steve Runnels, Chief Executive Officer of Myosana Therapeutics, Inc.

"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," said Nick Whitehead, Chief Scientific Officer of Myosana.

To learn more about PPMD's robust Research Strategy, funding initiatives, programmatic investments, and strategies for accelerating drug development, click here.

ABOUT PARENT PROJECT MUSCULAR DYSTROPHY:

Duchenne is a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne.

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. Follow PPMD on Facebook, Twitter, Instagram, and YouTube. 

ABOUT MYOSANA THERAPEUTICS, INC.:

Myosana Therapeutics, Inc. is a spin out from the University of Washington. Founders of the company are Stan Froehner and Nick Whitehead. Stan is the UW Medicine Distinguished Professor of Physiology & Biophysics in the School of Medicine at UW and also serves as the Chairman of Myosana Therapeutics. Nick is a Research Associate Professor in the department and his discovery for delivery of whole genes to skeletal and cardiac muscles using a non-viral platform have great potential to overcome many limitations of viral delivery. He also serves as CSO for Myosana. The initial focus of the Company is on disease-modifying therapeutics for Duchenne muscular dystrophy, but this therapeutic approach also opens the opportunity for treatment of other neuromuscular genetic diseases. Please see, www.myosanatherapeutics.com, for additional information.

SOURCE Parent Project Muscular Dystrophy (PPMD)

Related Links

http://www.parentprojectmd.org