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Notification report


Full notification file


General information

Notification Number
B/NL/14/008

Member State to which the notification was sent
Netherlands

Date of acknowledgement from the Member State Competent Authority
17/12/2014

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:
01/07/2014 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?
Yes:
Germany;

Has the same GMO been notified elsewhere by the same notifier?
No

GMO characterization

GMO is a:
DNA Virus

Identity of the GMO:
Parvoviridae
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