Full notification fileGeneral informationNotification NumberB/FR/15/GT04Member State to which the notification was sentFranceDate of acknowledgement from the Member State Competent Authority21/01/2015Title of the ProjectClinical 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 30/09/2015Name of the Institute(s) or Company(ies)Bluebird bio, Inc, ;
3. Is the same GMO release planned elsewhere in the Community?NoHas the same GMO been notified elsewhere by the same notifier?NoGMO characterizationGMO is a:Other: autologous CD34+ cellsIdentity 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.
Differentiation: Not ApplicableInformation relating to the recipient or parental organisms from wich the GMO is derived
European Commission administrative informationConsent given by the Member State Competent Authority:Not known