Explicação simples
Patients with this rare immune disorder have white blood cells that can catch bacteria but cannot kill them. PM359 takes the patient's own blood stem cells, uses prime editing to repair the exact mutation causing the fault, and returns them. In the first patients treated, the repaired cells took hold and the missing immune function came back.
Aprofundar
PM359 is an autologous CD34+ haematopoietic stem cell therapy in which prime editing corrects the NCF1 delGT mutation causing p47phox-deficient chronic granulomatous disease. Published results from the first two participants, following busulfan conditioning, reported prompt neutrophil and platelet engraftment, 69 and 83 per cent dihydrorhodamine-positive neutrophils by day 30, durable restoration of NADPH oxidase activity and early clinical benefit including resolution of CGD-associated colitis.
Sources
- New England Journal of Medicine · 2025
Prime Editing for p47phox-Deficient Chronic Granulomatous Disease ↗