Full notification file
Member State to which the notification was sent
Date of acknowledgement from the Member State Competent Authority
Title of the Project
A phase I/II, non-randomized, monocentric, open-label study of autologous CD34+ cells transduced with the G1XCGD lentiviral vector in patients with X-Linked Chronic Granulomatous Disease
Proposed period of release:
01/01/2015 to 31/12/2019
Name of the Institute(s) or Company(ies)
GENETHON, 1 bis rue de l’Internationale
3. Is the same GMO release planned elsewhere in the Community?
United Kingdom; Czech Republic;
Has the same GMO been notified elsewhere by the same notifier?
GMO is a:
Identity of the GMO:
Species: Homo Sapiens Sapiens
The GMO/IMP consists of patient-autologous CD34+ cells transduced ex vivo with the G1XCGD lentiviral vector containing the codon-optimized cDNA of the human CYBB gene for the treatment of X-linked Chronic Granulomatous Disease (CGD). The genetically-modified Investigational Medicinal Product (IMP) is intended to restore the NADPH-oxidase function in granulocytes and other phagocytes after hematopoietic reconstitution.
The cells will be used only for therapeutic purposes in the same patient from whom the cells were obtained (autologous application).
Information relating to the recipient or parental organisms from wich the GMO is derived
Human immunodeficiency virus (HIV)
European Commission administrative information
Consent given by the Member State Competent Authority: