Full notification file
Member State to which the notification was sent
Date of acknowledgement from the Member State Competent Authority
Title of the Project
AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects with Homozygous Familial Hypercholesterolemia (HoFH).
Proposed period of release:
01/01/2017 to 12/12/2018
Name of the Institute(s) or Company(ies)
University of Pennsylvania (UPenn), 3400 Spruce St.
8046 Maloney Building
Philadelphia, PA 19104-4283
United States of America;
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:
Species: Adeno-associated virus (AAV-derived-replication-deficient viral vector)
Information relating to the recipient or parental organisms from wich the GMO is derived
AAV2 (ITRs) /AAV8 (capsid)
European Commission administrative information
Consent given by the Member State Competent Authority: