A Phase I/II open-label, single-ascending-dose study of EN-374, a helper-dependent adenoviral-based gene therapy, in participants with X-linked chronic granulomatous disease
X-linked chronic granulomatous disease
A single dose of EN-374 administered by intravenous infusion after mobilization and followed by enrichment
1. Male 2. Age: ≥18 years at the time of signing the informed consent form (ICF) 3. Diagnosis of X-CGD with DHR+ cells ≤5% and a pathogenic mutation in the CYBB gene 4. History of at least one severe infection requiring medical intervention or chronic inflammatory disorder 5. Does not have a suitable, available, and willing human leukocyte antigen (HLA)-matched (10/10) related donor 6.EN-374 capsid total antibody titer below threshold 7. Use of highly effective contraception 8. Informed consent 9. Adequate organ function as indicated by the criteria in the Protocol
1. Active bacteremia or fungemia 2. History of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C 3. History or clinical evidence of any medical or social issues likely to put the participant at additional risk or to interfere with study conduct 4. History of HSCT or granulocyte transfusions 5. Known hypersensitivity to elements in the treatment regimen 6. Undergone investigational gene therapy 7. Treated with another investigational drug product within 30 days (or 5 half-lives) within 30 days before screening 8. Unable to comply with the visit and requirement of the protocol