Myosana is pioneering a targeted non-viral gene therapy platform for neuromuscular and cardiac genetic diseases. Our platform is designed to overcome the limitations of viral delivery, expanding the range of diseases that doctors can treat with gene therapy.
03-row-media-1AAV gene therapy works well for some conditions, but certain restrictions of AAV delivery make it unfeasible for other genetic diseases. AAV is limited in the size of genes it can deliver, which rules out the larger genes required to treat many diseases. AAV also triggers an immune reaction that makes it difficult for people with genetic conditions to tolerate repeated doses.
muscle-cs-1delivery, expanding the range of diseases that doctors can treat with gene therapy.
Our solution: A groundbreaking non-viral gene therapy platform that utilizes a protein called glucose transporter protein type-4 (GLUT4), which moves through muscle cells to deliver glucose, to deliver its genetic payload.
03-row-media-3It can carry genetic payloads of any size, expanding the potential of gene therapy to treat diseases with longer genetic makeups. Unlike viral vectors, the Myosana platform is designed to avoid an immune reaction, making repeat dosing possible—an essential advance for successful lifelong treatment of genetic diseases. In addition, the novel Myosana platform is the only truly muscle-directed non-viral gene therapy, allowing it to specifically target skeletal and cardiac muscle in patients with genetic diseases.