Full notification file
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?
Has the same GMO been notified elsewhere by the same notifier?
GMO is a:
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
Human Immunodeficiency Virus 1
Totipotent stem cells
Pluripotent hematopoietic stem cells
European Commission administrative information
Consent given by the Member State Competent Authority: