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
Evaluation of safety and efficacy of an intravenous injection of GNT0003, a suspension of recombinant AAV8 viral vector carrying the human UGT1A1 transgene, in patients with Crigler-Najjar syndrome.

Proposed period of release:
01/07/2017 to 01/12/2030

Name of the Institute(s) or Company(ies)
University of Amsterdam, Academic Medical Center of the University of Amsterdam;

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

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

GMO characterization

GMO is a:
DNA Virus

Identity of the GMO:
Order: Parvoviridae
Genus: Dependoparvovirus
Species: Adeno-associated virus (AAV)
Strain: the GMO is a pseudotyped AAV vector
ITRs present in the vector genome are derived from AAV serotype 2
The capsid consists of a capsid of AAV serotype 8
Common name: AAV2/8 or AAV8

Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
AAV2DependovirusAdeno‐Associated Virus---
AAV8DependovirusAdeno‐Associated Virus---

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known