Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
What this trial is testing
The first trial to use prime editing in people. Patients' own blood stem cells were corrected outside the body and returned. Both participants reported so far engrafted quickly and regained the immune function they had been missing, with early clinical improvement.
A Phase 1/2 study of PM359, an autologous prime-edited CD34+ cell therapy correcting the NCF1 delGT mutation. Published results in the first two participants, following busulfan conditioning, reported prompt neutrophil and platelet engraftment, 69 and 83 per cent dihydrorhodamine-positive neutrophils by day 30, and durable restoration of NADPH oxidase activity with early clinical benefit.
Sources
- New England Journal of Medicine · 2025
Prime Editing for p47phox-Deficient Chronic Granulomatous Disease ↗ - ClinicalTrials.gov
Study record NCT06559176 ↗
What a trial result does and does not mean
Early-phase results come from small numbers of participants and short follow-up. They can change with more people and more time, and a trial that is recruiting, active or even completed has not established that a treatment works. Enrolment criteria are strict; eligibility is decided by the trial site, never by a website.