GlobeNewswire·Jul 23, 2026, 4:05 PM ET

Prime Medicine Announces U.S. FDA Clearance of Investigational New Drug Application for PM577a in H1069Q-mutated Wilson Disease

PRME

FDA cleared the Investigational New Drug (IND) application for PM577a, allowing the company to proceed with clinical studies in the United States.
The IND clearance combined with a previously cleared New Zealand Clinical Trial Application (CTA) establishes a global Phase 1/2 program for the investigational in vivo Prime Editor.
PM577a targets the ATP7B H1069Q mutation, which is the most prevalent Wilson disease-causing allele in North America & Europe.
Prime Medicine expects to initiate the Phase 1/2 trial in the second half of 2026 & anticipates initial clinical data in 2027.
The open-label, first-in-human study will evaluate safety, tolerability, biological activity, & efficacy of ascending doses in adults & adolescents with Wilson disease.
Initial trial enrollment will focus on adults who are clinically stable on standard-of-care therapy, with assessments including copper efflux via 64 Cu PET & hepatic copper biopsy.
PM577a is an LNP-formulated Prime Editing product designed to correct pathogenic ATP7B mutations in hepatocytes via a single intravenous infusion.
The H1069Q allele accounts for approximately 30–50% of Wilson disease-associated variants in the U.S. & Europe, while a follow-on candidate targeting R778L is in pre-clinical development for East Asian populations.
Wilson disease is a rare genetic disorder affecting 1 in 30,000 individuals globally, currently treated with lifelong non-curative therapies or high-risk liver transplantation.
Prime Medicine utilizes its proprietary Prime Editing platform to develop one-time curative genetic therapies that aim to repair almost all types of genetic mutations.