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
Phase I/II safety, tolerability and initial efficacy study of adenoassociated viral vector serotype 9 containing human Sulfamidase gene after intracerebroventricular administration to patients with Mucolopolysaccharidosis IIIA (Sanfilippo A syndrome).

Proposed period of release:
30/09/2015 to 31/05/2016

Name of the Institute(s) or Company(ies)
Laboratorios del Dr. Esteve, S.A., Av. Mare de Déu de Montserrat, 221
08041 Barcelona;

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: Dependovirus
Species: Recombinant adeno-associated virus (AVV) non-replicative
The complete name of the recombinant AAV is AAV9-CAG-coh-SGSH (or AAV9-hSulfamidase).

Human recombinant adeno-associated vector serotype 9 (AAV9) containing human Sulfamidase is a breakthrough product for the treatment of Mucolopolysaccharidosis IIIA (Sanfilippo A Syndrome).

Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
AAV9DependovirusAdeno-associated Virus-Serotype 9-

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known