Myosana Therapeutics

Myosana Therapeutics, Inc. is pioneering efforts in developing non-viral targeted gene therapy for skeletal muscle and cardiac diseases. Our current focus is Duchenne muscular dystrophy (DMD) but our platform is applicable to any genetic muscle disease.

Current gene therapy for skeletal and cardiac muscle diseases uses Adeno Associated Virus (AAV) to deliver small healthy genes or portions of large genes, to the cells that contain unhealthy (mutated) genes. AAV has numerous limitations, including limited gene size capacity (5 kilobases), precluding its use for many larger genes like dystrophin. Furthermore, many individuals may have naturally occurring antibodies against AAV or will develop antibodies after their first treatment, limiting treatment to a single dose.

The Myosana Platform Technology bypasses the limitations of AAV delivery and, therefore, is much more versatile.

• The Myosana platform does not use viruses to deliver genes to the cells.

• The new technology can deliver genes of any size.

• The Myosana Non-Viral delivery method is much less likely to elicit an immune response, enabling repeated dosing over months or years.

• The Myosana platform targets the healthy gene to skeletal and cardiac muscle cells.

The novel gene therapy approach we have developed will provide new treatments for many diseases of skeletal and cardiac muscle. Our current focus is DMD, but we expect to use the platform to treat other muscular dystrophies and congenital myopathies.