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
An open-label, uncontrolled, single-dose, dose-escalation, multi-centre trial investigating the safety and efficacy of systemic administration of AAV5-hFIX, an adeno-associated viral vector containing a codon-optimised human factor IX gene, to severe haemophilia B

Proposed period of release:
01/01/2015 to 31/12/2015

Name of the Institute(s) or Company(ies)
uniQure Biopharma B.V., Meibergdreef 61
1105 BA Amsterdam
The Netherlands;

3. Is the same GMO release planned elsewhere in the Community?
Not known

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

GMO characterization

GMO is a:
DNA Virus

Identity of the GMO:
Genus: Dependovirus
Species: AAV-derived replication-deficient viral vector
The complete name of the vector is: Recombinant adeno-associated viral vector containing the codon-optimized human coagulation factor IX cDNA

Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
AAVDependovirusParvoviridaeAdeno-associated VirusN/ASerotype 5

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known