Prime Medicine Announces The New England Journal of Medicine Publication of PM359 Clinical Data for the Treatment of Chronic Granulomatous Disease

Core Insights - Prime Medicine, Inc. has published Phase 1/2 clinical data for PM359, an investigational autologous hematopoietic stem cell product for p47phox chronic granulomatous disease (CGD), in the New England Journal of Medicine [1][2] - The data indicates rapid neutrophil and platelet engraftment, durable restoration of NADPH oxidase activity, and early clinical benefits without safety concerns, marking the first-in-human demonstration of Prime Editing's safety and efficacy [2][3] Company Overview - Prime Medicine is focused on developing a new class of one-time curative genetic therapies using its proprietary Prime Editing platform, which aims to make precise edits in genes while minimizing unwanted modifications [4] - The company is advancing a diversified portfolio of therapeutic programs targeting liver, lung, immunology, and oncology diseases, with plans to expand into additional genetic and immunological diseases, cancers, and infectious diseases [5] Clinical Trial Details - The Phase 1/2 trial involved two patients with a history of CGD complications, both of whom showed significant improvements, including 69% and 83% dihydrorhodamine-positive neutrophils by Day 30, exceeding the 20% threshold for clinical benefit [6] - Both patients remained free of new CGD-related complications post-infusion, with one patient stopping mesalamine treatment without flare-ups, and the other showing decreased levels of fecal calprotectin [6]