Clinical Research Directory

Inclusion Criteria: * Male * ≥ 18 years of age during dose escalation, then ≥ 3 months of age during dose expansion * Diagnosis of X-CGD with DHR+ cells ≤ 5% and a pathogenic mutation in the CYBB gene * History of at least 1 severe infection requiring medical intervention or chronic inflammatory disorder * Does not have a suitable, available, and willing human leukocyte antigens (HLA)-matched (10/10) related donor * Non-sterile male participants who are or may become sexually active with female partners of childbearing potential are required to use highly effective contraception * Informed consent, with informed assent from capable participants * Adequate organ function Exclusion Criteria: * Active bacteremia or fungemia * History of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C * History or clinical evidence of any medical or social issues likely to put the participant at additional risk or to interfere with study conduct * History of HSCT or granulocyte transfusions * Known hypersensitivity to elements in the treatment regimen * Undergone investigational gene therapy * Treated with another investigational drug product within 30 days before screening * Unable to comply with the visits and requirements of the protocol as determined by the Investigator