An article in the New England Journal of Medicine (Volume 394, Issue 12, Pages 1229-1230) publishes data from a Phase 1/2 clinical trial of Prime Medicine's PM359 for the treatment of chronic granulomatous disease (CGD) caused by a p47phox mutation. PM359 is an investigational autologous hematopoietic stem cell (HSC) product. Data show rapid engraftment, 69% and 83% of DHR+ neutrophils in patients. Persistent NADPH oxidase activity and no new safety signals are observed. In patient 2, there was a significant decrease in fecal calprotectin. The results were also presented at the 67th ASH Annual Meeting (December 6-9, 2025). Prime Medicine uses the proprietary Prime Editing platform for precise gene editing without unwanted DNA changes.[1]