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
A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen Storage Disease Type Ia (GSDIa)”. Protocol Number 401GSDIA01.

Proposed period of release:
01/10/2018 to 31/12/2019

Name of the Institute(s) or Company(ies)
Ultragenyx Pharmaceutical, Inc., 840 Memorial Drive
Cambridge, MA 02139

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:
Genus: Dependoparvovirus
Species: Adeno-associated virus serotype 8 (AAV8)

Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
AAVDependoparvovirusAdeno‐Associated Virus--Serotype 8

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known