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Notification report

Full notification file


General information

Notification Number
B/IE/13/01

Member State to which the notification was sent
Ireland

Date of acknowledgement from the Member State Competent Authority
13/02/2013

Title of the Project
A Phase 1 safety study in subjects with severe Hemophilia B (Factor IX deficiency) using a single-stranded, adeno-associated pseudotype 8 viral vector to deliver the gene for human Factor IX [Protocol # AAV8-hFIX19-101]

Proposed period of release:
01/04/2013 to 30/09/2015

Name of the Institute(s) or Company(ies)
Children’s Hospital of Philadelphia, Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia
5th Floor Colket Translational Research Building
3501 Civic Center Boulevard
Philadelphia, PA 19104-4319
USA;


3. Is the same GMO release planned elsewhere in the Community?
No

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

GMO characterization

GMO is a:
DNA Virus

Identity of the GMO:
AAV8-hFIX19 is pseudotyped with AAV8 capsid proteins, containing a single-stranded DNA genome of 4275 nucleotides consisting of an hFIX expression cassette flanked by the 145 nucleotide inverted terminal repeats derived from AAV type 2. All of the viral coding sequences have been removed and replaced with the hFIX expression cassette.

Information relating to the recipient or parental organisms from wich the GMO is derived

Common Name	Genus	Species	Subspecies	Strain	Pathovar
Adeno-associated virus	Dependovirus	Adeno-associated virus (AAV)	-	-	AAV2/AAV8

European Commission administrative information

Consent given by the Member State Competent Authority:
Yes
03/07/2013 00:00:00
Remarks:
Consent granted subject to conditions