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-207 titled: “A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects with Transfusion-dependent β-Thalassemia, who do not have β0/β0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with a Lentiviral βA-T87Q-Globin Vector in Subjects ≥12 and ≤50 Years of Age”

Proposed period of release:
01/01/2016 to 31/12/2018

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

3. Is the same GMO release planned elsewhere in the Community?
France; United Kingdom; Greece; Italy;

Has the same GMO been notified elsewhere by the same notifier?

If yes, notification number(s):

GMO characterization

GMO is a:
RNA Virus

Identity of the GMO:
The GMO consists of the 2 key elements briefly described below: a recombinant lentivirus which transduces autologous haematopoietic stem cells. LentiGlobin BB305 lentiviral vector: a recombinant HIV-1 RNA lentivirus (rLV) manufactured with 4 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+ haematopoietic stem cells transduced with the LentiGlobin BB305 lentiviral vector.

Lineage: Totipotent stem cells

Differentiation: Pluripotent haematopoietic stem cells

Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
HIV-1LentivirusHuman Immunodeficiency Virus 1-HIV-1 NL4-3-
HumanHomoSapiensTotipotent stem cellsPluripotent haematopoietic stem cells-

European Commission administrative information

Consent given by the Member State Competent Authority:
15/12/2016 00:00:00