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.