Prime Medicine, Inc. has made a significant advancement in genetic therapies with the publication of Phase 1/2 clinical data concerning its investigational product, PM359, for treating p47phox chronic granulomatous disease (CGD). The findings were published in the prestigious New England Journal of Medicine on December 7, 2025, and will also be showcased at the 67th American Society of Hematology (ASH) Annual Meeting, taking place from December 6 to 9, 2025, in Orlando, Florida.
The publication, titled “Prime Editing for p47-phox Chronic Granulomatous Disease,” presents initial results from two patients involved in the Phase 1/2 trial. This trial was designed to evaluate the safety, biological activity, and preliminary efficacy of PM359 in both adult and pediatric participants. Notably, both patients exhibited rapid engraftment of neutrophils and platelets, alongside a durable restoration of NADPH oxidase activity. These results indicate early clinical benefits without any reported safety concerns.
This clinical trial represents the first human demonstration of the safety and efficacy of Prime Editing, a novel approach in genetic therapy. The outcomes support the potential for PM359 to serve as a precise therapeutic strategy for patients suffering from CGD, a rare genetic disorder that can lead to severe infections.
Prime Medicine’s commitment to developing a new class of differentiated one-time curative genetic therapies is underscored by these promising findings. The company’s focus on delivering transformative solutions for patients highlights the ongoing advancements in biotechnology and genetic medicine.
With the publication of these initial data, Prime Medicine aims to foster further interest and investment in its groundbreaking research and development efforts. As the field of genetic therapies continues to evolve, the implications of PM359’s success could pave the way for future treatments for various genetic conditions.
