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Notification report


Full notification file


General information

Notification Number
B/DE/17/PEI3281

Member State to which the notification was sent
Germany

Date of acknowledgement from the Member State Competent Authority
08/12/2017

Title of the Project
Clinical trial ALD-102 titled: "A phase 2/3 study of the efficacy and safety of hematopoietic stem cells transduced with Lenti-D lentiviral vector for the treatment of cerebral adrenoleukodystrophy (CALD)".

Proposed period of release:
01/12/2018 to 01/12/2019

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;

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:
Two GMOs are described below: Lenti D Drug Product and Lenti-D lentiviral vector.
Lenti D Drug Product: Lenti-D Drug Product consists of autologous CD34+ cell-enriched population that contains cells transduced with lentiviral vector that encodes an ABCD1 cDNA for human ALDP, suspended in cryopreservation solution in the final immediate container for the intended medical use.
Lineage: Totipotent stem cells
Differentiation: Pluripotent hematopoietic stem cells
Lenti D lentiviral vector: replication defective, self-inactivating (SIN) recombinant HIV-1 RNA lentiviral vector (rLV) manufactured with 5 recombinant plasmids designed to express all the packaging components to generate a modified rLV. The Lenti D lentiviral vector packaged RNA transcript encodes the therapeutic ABCD1 cDNA.
Lineage: HIV-1
Differentiation: Not Applicable


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-
HumanHomoSapiens---
MoMuLVGamma retrovirusMurine leukemia virus-Moloney-
VSVVesciculovirusVescicular Stomatitis Virus-Vesicular stomatitis Indiana virus-

European Commission administrative information

Consent given by the Member State Competent Authority:
Yes
18/06/2018 00:00:00
Remarks: