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 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?

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

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 NameGenusSpeciesSubspeciesStrainPathovar

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known