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Treatment · Chronic granulomatous disease

PM359

The first prime-editing therapy given to human patients, with published evidence that it restored the immune function those patients lacked.

Phase I/II prime editingex vivofirst-in-humanNCF1
Clinical research Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.

Explicación sencilla

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.

Profundizar

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

Written by The CRISPR Atlas editorial team Last updated Aug 25, 2026

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Technologies

Prime Editing

Diseases

Chronic Granulomatous Disease

Clinical Trials

A Study of the Safety and Efficacy of Prime Editing (PM359) in Participants With p47phox Autosomal Recessive Chronic Granulomatous Disease

Genes

NCF1

Companies

Prime Medicine

Research

Prime editing for p47phox-deficient chronic granulomatous disease
Solo información educativa Esta página es una referencia, no asesoramiento médico. El estado de la investigación y la situación regulatoria cambian; comprueba la fecha de última actualización que aparece arriba y confirma cualquier dato importante con las fuentes primarias indicadas.