{"id":"nct:NCT07748403","kind":"trial","title":"A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)","authors":["Prime Medicine, Inc."],"author_count":1,"year":2026,"published":"2026-08-05","venue":"ClinicalTrials.gov","cited_by":null,"doi":null,"arxiv_id":null,"nct_id":"NCT07748403","source_url":"https://clinicaltrials.gov/study/NCT07748403","archive_url":"https://space-kitty.com/papers/nct:NCT07748403","updated":"2026-10-01","pmid":null,"pmcid":null,"openalex_id":null,"pdf_url":null,"abstract":"The purpose of this study is to evaluate the safety, tolerability, biological activity, and initial efficacy of PM577a, an investigational Prime Editing therapy, in adults and adolescents with Wilson disease (WD). Wilson disease is caused by changes (mutations) in the ATP7B gene that prevent the body from removing excess copper normally. PM577a is designed to precisely correct one of the most common disease-causing ATP7B mutations (p.H1069Q) in liver cells with the goal of restoring normal copper metabolism. This is the first study of PM577a in people. Participants will receive a single intravenous (IV) infusion of PM577a and will be monitored closely to evaluate safety, how the body responds to treatment, whether copper metabolism improves, and whether treatment may improve signs and symptoms of Wilson disease.","abstract_license":"ClinicalTrials.gov (U.S. National Library of Medicine), data processed 2026-10-29T09:00:05; modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters","license":"ClinicalTrials.gov terms (attribution, processing date, modifications noted)","is_open_access":true,"retracted":false,"peer_reviewed":null,"categories":["Wilson Disease","Wilson's Disease","Wilsons Disease"],"details":{"acronym":"PM577a","status":"RECRUITING","phase":"PHASE1, PHASE2","study_type":"INTERVENTIONAL","enrollment":42,"sponsor":"Prime Medicine, Inc.","conditions":["Wilson Disease","Wilson's Disease","Wilsons Disease"],"interventions":["DRUG: PM577a"],"start_date":"2026-09-01","start_date_type":"ESTIMATED","primary_completion_date":"2028-11-01","has_results":false,"data_source":"ClinicalTrials.gov","data_processed":"2026-10-29T09:00:05","modifications":"selected fields only; whitespace normalized; brief summary truncated to 2,000 characters"},"provenance":"clinicaltrials","attribution":"Source: ClinicalTrials.gov (U.S. National Library of Medicine), data processed 2026-10-29T09:00:05; modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters. Not a U.S. Government endorsement.","topics":[{"slug":"gene-editing-therapies","name":"CRISPR & Gene-Editing Therapies","domain_slug":"medicine"}],"cited_in_dossiers":[]}