Notification report

Full notification file

General information

Notification Number

Member State to which the notification was sent

Date of acknowledgement from the Member State Competent Authority

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?

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 NameGenusSpeciesSubspeciesStrainPathovar
HIV-1LentivirusHuman Immunodeficiency Virus 1N/AHIV-1 NL4-3N/A
HumanHomoSapiensTotipotent stem cellsPluripotent hematopoietic stem cellsN/A

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known