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-104 (EudraCT No. 2018-001145-14) titled: “A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects ≤17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)”

Proposed period of release:
01/01/2020 to 30/09/2023

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

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

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

If yes, notification number(s):

GMO characterization

GMO is a:
Other: mammal

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

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.

Lineage: Totipotent stem cells

Differentiation: Pluripotent hematopoietic stem cells

Lenti D lentiviral vector: The Lenti D lentiviral vector (LVV) is a third generation, replication-defective, self-inactivating (SIN), human immunodeficiency virus 1 (HIV 1)-based LVV that is pseudotyped with the vesicular stomatitis virus envelope glycoprotein (VSV-G) and utilizes an internal MNDU3 promoter to control expression of the human ABCD1 transgene that encodes the human adrenoleukodystrophy protein (ALDP).

Lineage: HIV-1
Differentiation: Not Applicable

Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
HIV-1Lentiviridae…--HIV-1 NL4-3N/A
VSVVesciculovirusN/AN/AVesicular stomatitis Indiana virusN/A

European Commission administrative information

Consent given by the Member State Competent Authority:
18/06/2019 00:00:00