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Notification report


Full notification file


General information

Notification Number
B/IT/19/01

Member State to which the notification was sent
Italy

Date of acknowledgement from the Member State Competent Authority
11/02/2019

Title of the Project
AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects with Homozygous Familial Hypercholesterolemia (HoFH).

Proposed period of release:
01/04/2019 to 31/12/2020

Name of the Institute(s) or Company(ies)
REGENXBIO Inc., 9600 Blackwell Road
Suite 210
Rockville, MD 20850
United States of America;


3. Is the same GMO release planned elsewhere in the Community?
Yes:
Netherlands;

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

GMO characterization

GMO is a:
DNA Virus

Identity of the GMO:
Family: Parvoviridae
Genus: Dependoparvovirus
Species: Adeno-associated virus (AAV-derived-replication-deficient viral vector)


Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
Adeno-associated virusDependoparvovirusAdeno-associated dependoparvovirus--AAV2 (ITRs) /AAV8 (capsid)

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known