NON-VIRAL GENE THERAPY FOR MUSCLE DISEASES
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.
Muscle-Targeted, Non-Viral 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.
MYOSANA THERAPEUTICS LEADERSHIP TEAM
Chief Executive Officer
Prof. of Neurology
University of Washington
NICK WHITEHEAD, Ph.D.
Founder & Chief Science Officer
Nick trained as a muscle physiologist and has more than 20 years experience in DMD muscle research. He is currently a Research Associate Professor in the Department of Physiology and Biophysics at the University of Washington. His innovative Muscle-Targeted, Non-Viral platform has the potential to provide a novel gene therapy treatment for DMD a wide-range of other neuromuscular and cardiac disorders.
Justin has decades of neuromuscular disease research & developed Biglycan as a potential therapy for DMD. He is currently the Scientific Founder and SAB Chairman of Bolden Therapeutics.
MONKEL LEK, Ph.D.
Assistant Prof. of Genetics
STAN FROEHNER, Ph.D.
James is an expert in CMC drug development, with decades of experience in the Pharmaceutical industry. He is currently providing CMC advice for Myosana.
Monkel is an established researcher in the neuromuscular disease field and his research has led to novel gene discoveries for previously undiagnosed rare muscle diseases.
MATTHEW LUMLEY, M.D, Ph.D.
Matthew is a trained Cardiologist with a Ph.D. in cardiovascular physiology. He has extensive experience in the Bio-pharmaceutical field, with positions at Pfizer, as Medical Director of Rare Diseases and, more recently Moderna, where he was responsible for taking the first mRNA therapeutics for rare diseases into the clinic. Currently, Matthew is a Venture Partner at Medicxi.
Founder & Chairman
Stan has decades of experience in muscle research and discovered the Syntrophin proteins, members of the Dystrophin complex in muscle. He has extensive research experience in DMD. He is currently a Professor of Physiology and Biophysics at the University of Washington. Stan 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.
JEFF CHAMBERLAIN, Ph.D.
JAMES THOMPSON, Ph.D.
Head of CMC
Moderna Therapeutics (Retired)
SCIENTIFIC ADVISORY BOARD
Jeff is an internationally recognized leader in the gene therapy and muscular dystrophy fields and has been a pioneer in AAV micro-Dystrophin gene therapy research and clinical development for DMD.
Prof. of Medical Science