Our Platform

The Myosana platform has the potential to save lives and increase longevity, as well as improve quality of life for patients and their families who now face significant unmet needs in genetic disease therapy.

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Duchenne muscular dystrophy (DMD), is a severe genetic disorder that causes progressive muscle degeneration and weakness.

DMD affects about 200,000 individuals worldwide, almost exclusively males. Symptoms first appear in early childhood, followed by rapid deterioration of motor functions. By age 10 to 15, many young people with DMD are wheelchair-bound. DMD eventually compromises the heart and breathing muscles, leading to premature death from heart or respiratory failure.

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The novel Myosana platform is well suited for DMD because it is the only truly muscle-directed non-viral gene therapy.

In addition, it overcomes the limitations of traditional viral vector delivery that have made gene therapy for DMD unachievable.

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For transformative DMD treatment, we believe that gene therapy must deliver the full-length dystrophin gene.

However, the gene is too large to fit on a virus. Myosana’s large-capacity non-viral platform is the first platform capable of delivering the full-length dystrophin gene.

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Myosana’s platform is also mutation agnostic, making it applicable to patients with any mutation in the DMD gene.

It’s designed to avoid an immune reaction (a drawback of viral delivery), enabling repeat dosing for lifelong treatment.

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Successful treatment of DMD would not only transform the lives of DMD patients and their families.

It would also de-risk the entire Myosana platform, enabling us to rapidly pivot and target a wide range of neuromuscular disorders and cardiomyopathies.

 

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