Prime Medicine Publishes Phase 1/2 Clinical Data for PM359

Prime Medicine, Inc. has announced the publication of Phase 1/2 clinical trial data for its investigational therapy, PM359, aimed at treating p47phox chronic granulomatous disease (CGD). The findings appeared in the prestigious New England Journal of Medicine on December 7, 2025, showcasing promising results for patients involved in the study.

The publication, titled “Prime Editing for p47-phox Chronic Granulomatous Disease,” details the experiences of two patients treated with PM359. This investigational autologous hematopoietic stem cell product was evaluated for safety, biological activity, and preliminary efficacy in both adult and pediatric participants. Notably, both patients demonstrated rapid neutrophil and platelet engraftment along with a durable restoration of NADPH oxidase activity, which is crucial for immune function. There were no reported safety concerns, indicating a favorable profile for the treatment.

Promising Results and Future Implications

The data not only highlight the potential of PM359 as a precise therapeutic strategy for CGD but also mark a significant milestone in the application of Prime Editing technology in human subjects. The clinical results offer the first evidence of the safety and efficacy of this innovative approach, underscoring the potential for transformative impact on the treatment of genetic disorders.

These findings will also be featured in a poster session at the 67th Annual Meeting of the American Society of Hematology, scheduled from December 6 to 9, 2025, in Orlando, Florida. This event is a key gathering for professionals in the field, providing an opportunity to discuss advancements and ongoing research in hematology.

As the biotechnology sector continues to evolve, the results from Prime Medicine’s clinical trial represent a significant step forward in developing one-time curative therapies. The company remains committed to advancing its pipeline and exploring the full potential of Prime Editing for various genetic conditions.

The publication in a highly regarded journal like the New England Journal of Medicine not only validates the research efforts of Prime Medicine but also enhances its credibility within the scientific community. With continued focus on safety and efficacy, PM359 may pave the way for new treatments that could change the lives of patients suffering from CGD and similar genetic disorders.