Prime Medicine Doses First Patient in Trial of Investigational Wilson Disease Treatment

MT newswire2026/10/05 13:14

09:14 AM EDT, 10/05/2026 (MT Newswires) -- Prime Medicine (PRME) has dosed its first patient in a phase 1/2 clinical trial of PM577a, an investigational in vivo treatment for H1069Q-mutated Wilson disease, the company said Monday. The study will initially enroll adults who are clinically stable and being treated with standard-of-care therapy, the company said. Initial clinical data are expected next year, the company added. The biotechnology company also said the US Food and Drug Administration has granted Rare Pediatric Disease designation to PM577 for Wilson disease. Wilson disease is a rare, progressive genetic disorder which leads to copper accumulation in the liver, followed by damage to other organs, the company said.

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