Prime Medicine Publishes Positive PM359 Clinical Data in NEJM

Prime Medicine, Inc. has announced the publication of significant clinical data regarding its investigational therapy PM359 for treating p47phox chronic granulomatous disease (CGD). This data was published in the New England Journal of Medicine (NEJM) on December 7, 2025. The findings detail the outcomes of a Phase 1/2 trial, which evaluated both the safety and preliminary efficacy of PM359, an autologous hematopoietic stem cell product.

The published study, titled “Prime Editing for p47-phox Chronic Granulomatous Disease,” showcases results from two patients who participated in the trial. According to the data, both individuals exhibited rapid engraftment of neutrophils and platelets, along with a durable restoration of NADPH oxidase activity. Notably, these positive outcomes occurred without any reported safety concerns.

These findings offer the first human evidence supporting the efficacy and safety of Prime Editing technology in treating CGD. This development suggests that PM359 may represent a precise therapeutic approach for individuals suffering from this genetic condition.

The data will also be highlighted in a poster session at the upcoming 67th American Society of Hematology (ASH) Annual Meeting, taking place from December 6 to 9, 2025, in Orlando, Florida. This event will provide a platform for further discussion and dissemination of the research findings among healthcare professionals and researchers in the field.

The results of the PM359 study reflect Prime Medicine’s commitment to advancing novel genetic therapies aimed at providing one-time curative solutions for patients with serious health conditions. As the landscape of genetic medicine evolves, the implications of PM359 could lead to significant improvements in the management of CGD, offering hope for patients and their families.

With ongoing research and a focus on patient-centered outcomes, Prime Medicine is poised to contribute to the future of genetic therapies and the treatment of rare diseases.