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PM577a

Gene therapy · Gene replacement: ATP7B · developed by Prime Medicine

Phase 1/2 Most advanced trial

PRME Developer

1 Registered trials

What is PM577a?

PM577a is a gene therapy being evaluated for Wilson disease, a genetic disorder caused by mutations in the ATP7B gene that impairs copper transport. The therapy is designed to deliver a functional ATP7B gene to restore copper metabolism in patients with biallelic ATP7B variants.

Summary written with AI from the sources listed at the end of this page.

At a glance

Drug type: Gene therapy
Targets: ATP7B
Mechanism: Gene replacement: ATP7B
Therapy area: Gastroenterology, Neurology
Developer: Prime Medicine, Inc. (PRME)
FDA status: Investigational, not approved in the US. Most advanced trial: Phase 1/2

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT07748403	Phase 1/2	Wilson Disease	Recruiting	PRME

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Wilson DiseaseWilson's DiseaseWilsons Disease

More from Prime Medicine

PG-011 Phase 3 PG-011Gel Phase 3 PG-033 Phase 1 PM359 Phase 1/2 Pumecitinib Phase 2

Sources

ClinicalTrials.gov NCT07748403

Updated Oct 3, 2026. Something wrong or missing? Report it on the PRME page.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z