Therapeutic Programs

Facioscapulohumeral Muscular Dystrophy (FSHD)

FSHD is one of the most common muscular dystrophies, with over 40,000 patients in the U.S. There are no FDA approved therapies for this disease.

MC-DX4 aims to stop disease progression by eliminating expression of the toxic DUX4 protein in muscle tissue.

Head & Neck Squamous Cell Carcinoma (HNSCC)

Head & Neck cancer is the 6th most common form of cancer worldwide. For many patients, survival is measured in months, not years.

MC-30 aims to give years of quality life back to patients, by correcting an underlying mutation that creates resistance to current therapies.