NON-VIRAL GENE THERAPY FOR MUSCLE DISEASES
We are Passionate Innovators
Myosana Therapeutics, Inc. is leading the efforts in developing new gene therapies that will slow skeletal muscle degeneration and heart failure to improve the quality of life, increase longevity and reduce the disease burden of Duchenne muscular dystrophy (DMD) and other neuromuscular diseases.
Duchenne muscular dystrophy (DMD) is a severe genetic disorder characterized by progressive muscle degeneration and consequent muscle weakness. As a recessive, X-linked condition, it almost exclusively affects males – with approximately 200,000 individuals affected worldwide. DMD starts to show its effects during early childhood. The boy’s motor functions rapidly deteriorate with disease progression and, by only 10 to 15 years of age, many boys with DMD are wheelchair-bound. Eventually the heart and breathing muscles are compromised, leading to a premature death from heart or respiratory failure.
Limitations of Viral Vectors
Currently, Gene Therapy for muscle diseases (skeletal & cardiac) has Significant Limitations;
Adeno-Associated Virus (AAV) is a common virus used to deliver "fragments" of healthy genes to the cells that contain unhealthy (mutated) genes.
AAV has a limited gene size capacity of 5 kilobases (kb), precluding its use for many larger genes. As an example, Dystrophin, the gene responsible for Duchenne muscular dystrophy (DMD) is 14 kb, meaning that only one-third of the dystrophin gene can be "packaged" into AAV. These ‘micro-dystrophins’ might provide only partial improvement of muscle function.
AAV is most often restricted to a single administration due to the likelihood of an immune response to the virus. Also, many people already have preexisting immunity to AAV, which may prevent them from ever receiving this gene therapy.
AAV is not specifically targeted to muscle, so high doses are required to achieve delivery throughout the body. This may lead to dangerous side effects.
It is difficult and costly to manufacture large quantities of AAV.
A Non-Viral Gene Delivery Platform
We have developed a Platform Technology with key features to address the problems posed by AAV administration;
Our technology does not use viruses to deliver genes to the cells.
A Non-Viral delivery method is much less likely to elicit an immune response, enabling repeated dosing over months or years.
We are able to directly target the muscle cells.
We have developed antibodies to a specific muscle protein, which binds to the cell and delivers the appropriate gene into skeletal & cardiac muscle. This unique technology has application to a wide range of genetic diseases affecting skeletal and/or cardiac muscle.
Importantly, our platform is Not Restricted By Gene Size.
Full-Length Gene Delivery
Our Platform Technology consists of proprietary components that are designed to stabilize and effectively deliver full-length genes into muscles.
We have developed several monoclonal antibodies against a specific muscle target protein that is present in both skeletal and cardiac muscle.
We have developed other critical functions to ensure proper gene delivery. One component condenses the DNA of the gene and protects it from degradation. Another component provides stability in the circulation and assists in movement from blood vessels to the muscle. A third component provides a linking role that helps to deliver the DNA to the nucleus of the muscle cells.
THE MYOSANA THERAPEUTICS TEAM
Steve has more than twenty-eight years of successful and proven international business management experience in the healthcare industry. He has held the position of President and CEO of several start-up biopharmaceutical companies, in the US and Internationally. He was Executive Vice President and Board member of publicly traded NeoTherapeutics, Inc. (NEOT: NASDAQ) and Vice President of Marketing and Business Development at Sigma-Aldrich. He holds a B.S. in Cell Biology and certification from the American Society of Clinical Pathology as a specialist in Immunohematology.
NICK WHITEHEAD, Ph.D.
Co-Founder & CSO
Nick has more than 20 years experience in muscle research. He trained as a Physiologist in Australia, completing his Ph.D. at Monash University and a Postdoctoral Fellowship at the University of Sydney. He is currently a Research Associate Professor at the University of Washington. His research findings have pioneered important new insights into the mechanisms leading to muscle weakness in Duchenne muscular dystrophy (DMD). Nick has developed novel therapies that have shown great promise in pre-clinical models of Duchenne muscular dystrophy. His innovative Non-Viral platform has the potential to provide gene therapy treatment for a wide-range of neuromuscular disorders and other muscle-related diseases.
STAN FROEHNER, Ph.D.
Co-Founder and Chairman
Stan has decades of experience in muscle research and discovered the Syntrophin proteins, which form part of the Dystrophin complex in muscle. He has extensive research experience in DMD and has developed several pre-clinical treatments for the disease. Stan has received many accolades and was elected to the Washington State Academy of Sciences in 2013 and selected as a Fellow of the American Association for the Advancement of Sciences in 2017. Stan is currently Professor and Chair of the Department of Physiology and Biophysics at the University of Washington.
MIN JEONG KIM, Ph.D.
Min Jeong received her Ph.D. degree in the Department of Life Science at Ajou University in South Korea. She studied how syntrophin proteins regulate muscle cell differentiation and regulation. This research identified a novel role for syntrophin in muscle differentiation and migration, which are important processes in muscle regeneration after injury. Currently, she is testing novel therapeutic approaches to improve skeletal and cardiac function in DMD and has an integral role in the development of our non-viral gene delivery platform.