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Notification report

Full notification file


General information

Notification Number
B/FR/12/GT04

Member State to which the notification was sent
France

Date of acknowledgement from the Member State Competent Authority
01/08/2012

Title of the Project
Clinical trial HGB-205 titled: "A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the  Hemoglobinopathies (Sickle Cell Anemia and " Thalassemia Major) by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with a Lentiviral "A-T87Q Globin Vector (LentiGlobin® BB305 Drug Product)".

Proposed period of release:
01/01/2013 to 30/06/2016

Name of the Institute(s) or Company(ies)
Bluebird bio, Inc, ;


3. Is the same GMO release planned elsewhere in the Community?
Not known

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

GMO characterization

GMO is a:
RNA Virus

Identity of the GMO:
The GMO consists of the two key elements briefly described below: a recombinant lentivirus which transduces autologous hematopoietic stem cells.
LentiGlobin BB305 lentiviral vector: a recombinant HIV-1 RNA lentivirus (rLV) manufactured with 4 recombinant plasmids designed to express all the packaging components to generate a modified rLV. LentiGlobin BB305 lentiviral vector packaged RNA transcript encodes for the therapeutic βA-T87Q-globin gene.
LentiGlobin BB305 Drug Product: autologous CD34+ hematopoietic stem cells transduced with the LentiGlobin BB305 lentiviral vector.
Lineage: Totipotent stem cells
Differentiation: Pluripotent hematopoietic stem cells

Information relating to the recipient or parental organisms from wich the GMO is derived

Common Name	Genus	Species	Subspecies	Strain	Pathovar
HIV-1	Lentivirus	Human Immunodeficiency Virus 1	N/A	HIV-1 NL4-3	N/A
Human	Homo	Sapiens	Totipotent stem cells	Pluripotent hematopoietic stem cells	N/A

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known