Cell therapy for rare disease

Country

United States

An allogeneic cell therapy has been successfully used to treat patients 10 years and older with Duchenne muscular dystrophy, a rare genetic disease that affects both skeletal and cardiac muscle. The therapy, CAP-1002, was tested in a Phase 2, placebo-controlled trial and effectively reduced the deterioration of upper limb function in the patients as well as improved cardiac function. The results of the study, called HOPE-2, were published in The Lancet on 12 March 2022.