Prime Medicine, Inc. has announced the publication of its Phase 1/2 clinical trial data for PM359, an investigational autologous hematopoietic stem cell therapy aimed at treating p47phox chronic granulomatous disease (CGD). The results were published in the New England Journal of Medicine and represent a significant step forward in the development of one-time curative genetic therapies.
The publication, titled “Prime Editing for p47-phox Chronic Granulomatous Disease,” details the outcomes of two patients involved in the trial, which evaluated the safety, biological activity, and preliminary efficacy of PM359 in both adult and pediatric participants. During the trial, both individuals exhibited rapid engraftment of neutrophils and platelets. They also showed a durable restoration of NADPH oxidase activity, which is crucial for immune response, and experienced early clinical benefits without any reported safety concerns.
These findings provide the first demonstration of the safety and efficacy of Prime Editing in human subjects. The data suggests that PM359 could serve as a precise therapeutic strategy for CGD, a rare genetic disorder that impairs the body’s ability to combat infections.
Presentation at ASH Annual Meeting
In addition to the publication, the clinical data will be showcased during a poster session at the 67th American Society of Hematology (ASH) Annual Meeting, taking place from December 6 to 9, 2025, in Orlando, Florida. This event will provide an opportunity for researchers and clinicians to engage with the findings and discuss their implications for future therapies.
The results from the PM359 trial represent a significant milestone for Prime Medicine, a company dedicated to developing innovative genetic therapies. With the publication in such a prestigious journal and the presentation at a major conference, the company aims to raise awareness about the potential of Prime Editing technology in treating genetic disorders.
As ongoing research continues, the medical community will be watching closely for further developments concerning PM359 and its application in clinical settings. The initial outcomes are promising, and they may pave the way for new treatment options for patients suffering from CGD and similar conditions.
