Full notification file
Member State to which the notification was sent
Date of acknowledgement from the Member State Competent Authority
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
3. Is the same GMO release planned elsewhere in the Community?
Has the same GMO been notified elsewhere by the same notifier?
GMO is a:
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
Adeno-associated virus (AAV)
European Commission administrative information
Consent given by the Member State Competent Authority:
Consent granted subject to conditions