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Notification report

Full notification file


General information

Notification Number
B/ES/14/09

Member State to which the notification was sent
Spain

Date of acknowledgement from the Member State Competent Authority
04/11/2014

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?
No

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: 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 Name	Genus	Species	Subspecies	Strain	Pathovar
AAV9	Dependovirus	Adeno-associated Virus	-	Serotype 9	-

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known