Prime Medicine Announces U.S. FDA Clearance of Investigational New Drug Application for PM577a in H1069Q-mutated Wilson Disease
— FDA clearance of the IND, together with the previously cleared CTA, establishes a global Phase 1/2 clinical program for PM577a —
— PM577a targets the H1069Q mutation in the ATP7B gene, the most prevalent WD-causing allele in North America and Europe —
— Initial clinical data expected in 2027 —
Excerpt from the Press Release:
CAMBRIDGE, Mass., July 23, 2026 (GLOBE NEWSWIRE) — Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for PM577a, an investigational in vivo Prime Editor for Wilson disease (WD). With the IND cleared, PM577a may proceed to clinical study in the United States. Together with the Company’s previously announced New Zealand Clinical Trial Application (CTA) clearance, the IND clearance establishes a global Phase 1/2 program and opens participation to patients in the United States, where H1069Q is the single most common pathogenic variant causing WD.
“Wilson disease is a serious, progressive disorder with no approved curative option, and today’s standard of care requires lifelong therapy burdened by significant side effects and low adherence,” said Allan Reine, M.D., Chief Executive Officer of Prime Medicine. “With PM577a, we have the potential to offer a one-time therapy that corrects the disease at its genetic root. This clearance, alongside our recent New Zealand CTA, establishes a global Phase 1/2 program that reflects Prime’s confidence in our in vivo Prime Editing approach. We expect to initiate the Phase 1/2 trial in the second half of 2026 with initial clinical data anticipated in 2027, and we are committed to advancing this program with the rigor patients deserve.”
Phase 1/2 Clinical Trial
The Phase 1/2 clinical trial is an open-label, global, first-in-human study designed to evaluate the safety, tolerability, biological activity, and efficacy of ascending doses of PM577a in adults and adolescents with WD.
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