Prime Medicine doses first patient in nanoparticle-delivered gene-editing trial for Wilson disease
Published in Drugs development & discovery, Nanomedicine, Personalized medicine.
Published October 5, 2026. Source date: October 5, 2026.
Prime Medicine has dosed the first patient in its global Phase 1/2 trial of PM577a, an investigational Prime Editing therapy for Wilson disease associated with the ATP7B H1069Q mutation.
The treatment uses lipid nanoparticles to deliver gene-editing components to liver cells through a single intravenous infusion. It is designed to correct the underlying mutation rather than provide ongoing management of copper accumulation.
The open-label, first-in-human study will evaluate ascending doses for safety, tolerability and preliminary efficacy. It initially enrolls adults clinically stable on standard treatment; the broader study includes adults and adolescents with at least one H1069Q allele. Initial clinical data are expected in 2027.
The company also reported FDA Rare Pediatric Disease designation. That designation and first dosing do not establish efficacy, a cure, or marketing approval. The announcement contains no patient outcome results.
Source: Prime Medicine announcement. Trial identifier: NCT07748403.