Prime Medicine says it gave PM577a to a Wilson disease patient on October 5, in the first reported clinical use of prime editing to change DNA directly inside a person, but no results yet show whether it changed the patient’s DNA or improved their health.
The company’s worldwide Phase 1/2 trial is testing the experimental treatment in people with a disease-causing H1069Q mutation in the ATP7B gene, which interferes with how the body handles copper. When copper builds up, it can damage the liver and nervous system. Unlike methods that take cells out of the body, edit them, and put them back, PM577a is designed to carry DNA-editing tools to liver cells through one infusion into a vein.
The study will check safety, how well people tolerate treatment, its effects in the body, and early signs that it works as doses increase. It plans to include adults and adolescents, starting with adults whose condition is stable on standard treatment. Prime editing is designed to make specific changes to DNA.
The treatment remains experimental. The intended correction has not been publicly confirmed in this patient. The company expects to report initial clinical data in 2027.