Prime Medicine registers Phase 1/2 trial of PM577a prime editing therapy for Wilson disease
A newly listed trial will test a single infusion of Prime Medicine's investigational gene editing therapy in patients with a specific ATP7B mutation.
Prime Medicine had one new trial listing on the registry this week.
- The Phase 1/2 study (NCT07748403) is recruiting participants to test PM577a, described as the first study of PM577a in people, in which participants will receive a single intravenous infusion and be monitored for safety, treatment response, copper metabolism changes, and possible symptom improvement.1 The therapy targets Wilson disease patients carrying at least one allele with the p.H1069Q mutation in the ATP7B gene1, and it is designed to precisely correct one of the most common disease-causing ATP7B mutations in liver cells with the goal of restoring normal copper metabolism1. The study plans to enroll 42 participants1, with an estimated start in September 2026 and primary completion expected in November 2028.
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