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Notification report


Full notification file


General information

Notification Number
B/ES/18/02

Member State to which the notification was sent
Spain

Date of acknowledgement from the Member State Competent Authority
26/04/2018

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
USA;


3. Is the same GMO release planned elsewhere in the Community?
No

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

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