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Notification report


Full notification file


General information

Notification Number
B/NL/19/006

Member State to which the notification was sent
Netherlands

Date of acknowledgement from the Member State Competent Authority
20/05/2019

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 31/12/2020

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


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

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 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
HumanHomoSapiens---
MoMuLVGamma retrovirusN/AN/AMoloneyN/A
VSVVesciculovirusN/AN/AVesicular stomatitis Indiana virusN/A

European Commission administrative information

Consent given by the Member State Competent Authority:
Yes
22/11/2019 00:00:00
Remarks: