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Notification report

Full notification file


General information

Notification Number
B/FR/15/GT05

Member State to which the notification was sent
France

Date of acknowledgement from the Member State Competent Authority
09/04/2015

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
91002 Evry
France;


3. Is the same GMO release planned elsewhere in the Community?
Yes:
United Kingdom; Czech Republic;

Has the same GMO been notified elsewhere by the same notifier?
No

GMO characterization

GMO is a:
Other: Mammal

Identity of the GMO:
Genus: Human
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

Common Name	Genus	Species	Subspecies	Strain	Pathovar
Lentiviral vectors	Lentivirus	Human immunodeficiency virus (HIV)	HIV-1

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known