Notification report

Full notification file

General information

Notification Number

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:
01/04/2019 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?

Has the same GMO been notified elsewhere by the same notifier?

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)

Final report

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known