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Notification report

Full notification file


General information

Notification Number
B/FR/15/GT04

Member State to which the notification was sent
France

Date of acknowledgement from the Member State Competent Authority
21/01/2015

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 childhood cerebral adrenoleukodystrophy (CCALD)”.

Proposed period of release:
01/09/2013 to 30/09/2015

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


3. Is the same GMO release planned elsewhere in the Community?
No

Has the same GMO been notified elsewhere by the same notifier?
No

GMO characterization

GMO is a:
Other: autologous CD34+ cells

Identity of the GMO:
Two GMOs are described below: Lenti-D Drug Product and Lenti-D lentiviral vector.

Lenti-D Drug Product: autologous CD34+ hematopoietic stem cells transduced with the
Lenti-D lentiviral vector.
Lineage: Totipotent stem cells
Differentiation: Pluripotent hematopoietic stem cells

Lenti-D lentiviral vector: The Lenti-D lentiviral vector is a replication defective, selfinactivating
(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 Name	Genus	Species	Subspecies	Strain	Pathovar
-	-	-	-	-	-

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known