Jennifer L. Gori, Élie Haddad, Haydar Frangoul, Donald B. Kohn, Emma Morris, Bradley N. Martin, Briana Deary, Mike Nickerson, Rebecca L. Scholz, Isabel Fernández, Karine Léveillé, Suk See De Ravin, Elizabeth M. Kang, Marie Pierzynski, Tyra Estwick, Patricia Littel, Douglas B. Kuhns, Debra A. Long Priel, Pierre Teira, Stuart E. Turvey, Jordyn Arnold, Misty Evans, Meghann McManus, Ben Carpenter, David P. Waterman, Andrew V. Anzalone, Alexandria Petrusich, Christa E. Osuna, Tiernan T. O’Malley, Jacob Stewart-Ornstein, Jack Heath, Barrett J. Nehilla, Mohammed Asmal, Harry L. Malech
. We developed PM359, an autologous CD34+ hematopoietic stem-cell therapy in which prime editing is used to correct delGT. Two participants received PM359 after myeloid conditioning with busulfan: neutrophils and platelets engrafted promptly in both patients. Adverse events were consistent with myeloid conditioning with busulfan. NADPH oxidase activity was observed in neutrophils within 1 month and was maintained for 6 months and 4 months as of the last follow-up visit in Participants 1 and 2, respectively. These results support further investigation of prime editing of CD34+ cells to treat p47-CGD. (Funded by Prime Medicine; ClinicalTrials.gov number, NCT06559176.).