Full notification fileGeneral informationNotification NumberB/ES/14/09Member State to which the notification was sentSpainDate of acknowledgement from the Member State Competent Authority04/11/2014Title of the ProjectPhase 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 31/05/2016Name of the Institute(s) or Company(ies)Laboratorios del Dr. Esteve, S.A., Av. Mare de Déu de Montserrat, 221
3. Is the same GMO release planned elsewhere in the Community?NoHas the same GMO been notified elsewhere by the same notifier?NoGMO characterizationGMO is a:DNA VirusIdentity of the GMO:Family: Parvoviridae
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
European Commission administrative informationConsent given by the Member State Competent Authority:Not known
|AAV9||Dependovirus||Adeno-associated Virus||-||Serotype 9||-|