PM577

PM577 is a gene editing therapy in preclinical development by Prime Medicine for the treatment of Wilson's disease, specifically targeting patients with the most prevalent mutation in the United States. The therapy utilizes Prime Editing technology to precisely correct mutations in the ATP7B gene, which encodes a copper-transporting ATPase essential for copper homeostasis. Mutations in ATP7B cause Wilson's disease, an autosomal recessive disorder characterized by toxic accumulation of copper primarily in the liver and brain. By correcting these mutations at the DNA level, PM577 aims to restore normal function of ATP7B and provide a one-time curative genetic therapy for affected individuals[1][3][4][9].

Other names

Wilson’s Disease treatment (Prime Medicine)