Prime Medicine Doses First Patient in Wilson Disease Trial

Prime Medicine has dosed the first patient in a global Phase 1/2 clinical trial of PM577a for H1069Q-mutated Wilson disease, marking the company's first-ever human study of an in vivo Prime Editing therapy. Shares rose following the announcement as the biotechnology firm advanced its lead liver-directed program into clinical evaluation.

"Dosing the first patient with PM577a is a meaningful step toward our goal of delivering a transformative, one-time treatment for people living with Wilson disease," said Allan Reine, M.D., Chief Executive Officer of Prime Medicine.

Trial Design and Regulatory Status

The open-label, first-in-human study, registered under ClinicalTrials.gov identifier NCT07748403, evaluates ascending doses of PM577a in adults and adolescents. Eligible participants must carry at least one p.H1069Q allele in the ATP7B gene, with the initial cohort enrolling adult patients who remain clinically stable on standard-of-care therapies.

The U.S. Food and Drug Administration granted PM577 Rare Pediatric Disease designation to support development. Prime Medicine reported that initial clinical data from the trial are expected in 2027.

Understanding Wilson Disease and Treatment Limitations

Wilson disease is a rare, progressive genetic disorder caused by mutations in the ATP7B gene, leading to toxic copper accumulation primarily in the liver and brain. Standard management relies on lifelong chelation therapy or dietary restrictions, which often present tolerability and adherence hurdles for patients.

"Wilson disease is a serious, progressive genetic disorder with no approved curative therapy. Current pharmacologic treatments require lifelong management and can be limited by tolerability and adherence challenges," said Mohammed Asmal, M.D., Ph.D., Chief Medical Officer of Prime Medicine.

Unlike traditional gene therapies that add a functional gene copy without correcting the host DNA, Prime Editing functions as a search-and-replace word processor for genomes. The investigational PM577a treatment aims to directly correct the H1069Q mutation at the DNA level within liver cells to restore normal copper metabolism.

Market Response and Financial Standing

In public trading following the clinical milestone, Prime Medicine Inc (PRME) shares rose 11.45% to close at $3.99 compared to the previous session's close of $3.58, according to Finnhub market data as of October 9, 2026.