Advancing Novel Treatments for Patients with Muscle Disease

Standard of Care / Unmet Need

Duchenne muscular dystrophy (DMD) is a fatal genetic disorder that causes progressive weakness and degeneration of muscle. Because DMD is X-linked, it mainly affects boys and young men. Muscle weakness usually becomes evident in early childhood. Most patients are unable to walk by their early teens, and cardiomyopathy develops by the late teens, leading to death due to cardiac or respiratory failure by early adulthood. Duchenne affects about 1 in 3,500-5,000 live male births, and about 20,000 children are diagnosed globally each year.

Duchenne is caused by mutations in DMD gene encoding dystrophin, a protein that protects muscle cells from damage during contraction by connecting the cytoskeleton to the extracellular matrix, stabilizing the cell membrane. Mutations that reduce the amount or function of dystrophin leave muscle cells vulnerable to damage during contraction, leading to muscle damage, inflammation, and eventually permanent muscle loss and replacement with non-contractile fibrous tissue and fat.

Our Approach

Kate Therapeutics is combining its capsid and cargo technologies to deliver a shortened version of dystrophin (microdystrophin) potently and uniformly to skeletal muscle and heart, while avoiding off-target tissues like the liver, supporting the potential to be a one-time safe and effective gene therapy.

DMD Resources