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Notification report


Full notification file


General information

Notification Number
B/DE/17/PEI2978

Member State to which the notification was sent
Germany

Date of acknowledgement from the Member State Competent Authority
09/01/2017

Title of the Project
A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects with Transfusion-dependent β-Thalassemia, who have a β0/β0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with a Lentiviral βA-T87Q-Globin Vector in Subjects ≤50 Years of Age

Proposed period of release:
01/01/2017 to 31/12/2019

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


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

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

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
HumanHomoSapiens---

European Commission administrative information

Consent given by the Member State Competent Authority:
Yes
24/07/2017 00:00:00
Remarks: