Summary Notification

Full notification file

General information

Notification Number
B/DE/13/PEI1863

Member State to which the notification was sent
Germany

Date of acknowledgement from the Member State Competent Authority
29/04/2013

Title of the Project
Phase I/II Clinical Study of autologous CD34+ cells transduced ex vivo with the G1XCGD lentiviral vector containing the GP91 transgene which is a codon-optimized cDNA of the human CYBB gene (also known as GP91-PHOX gene) for the Treatment of X-linked chronic granulomatous disease (G1XCGD).

Proposed period of release:
01/10/2013 to 30/06/2018

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:
Germany; France; United Kingdom;

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

GMO characterization

GMO is a:
Viroid

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-oxydase 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:
Yes
13/11/2013 00:00:00
Remarks:

Notification report