PM577a

PM577a is a liver-targeted gene editing therapy being developed by Prime Medicine for the treatment of Wilson Disease. It utilizes Prime Editing technology, delivered via lipid nanoparticles (LNPs), to precisely correct the pathogenic p.H1069Q mutation in the ATP7B gene. By restoring the wild-type sequence at the endogenous genomic locus without inducing double-strand DNA breaks, PM577a aims to restore native gene regulation, ATP7B protein function, and copper homeostasis. Preclinical data in humanized mouse models have demonstrated efficient gene correction and normalization of hepatic copper levels, with durable effects observed in non-human primates.