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Full notification Final report - General information Notification Number B/ES/21/16 Member State to which the notification was sent Spain Date of acknowledgement from the Member State Competent Authority 09/06/2021 Title of the Project A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Seamless, Adaptive,Safety, Dose-Finding, and Phase 3 Clinical Study of UX701 AAV-Mediated GeneTransfer for the Treatment of Wilson Disease. Proposed period of release: 01/03/2022 to 31/05/2026 Name of the Institute(s) or Company(ies) Ultragenyx Pharmaceutical, Inc., 60 Leveroni Ct Novato, CA 94949 USA
Is the same GMO been notified elsewhere by the same notifier? Yes:
Austria; Belgium; Germany; Denmark; Finland; France; Italy; Portugal; Has the same GMO been notified elsewhere by the same notifier? No GMO characterization GMO is a: DNA Virus Identity of the GMO: Genus: DependoparvovirusSpecies: Recombinant adeno-associated viral vector derived from naturally occurring AAV9serotype Information relating to the recipient or parental organisms from which the GMO is derived Common Name: N/A Genus: Dependoparvovirus Species: Adeno-associated dependoparvovirus A Subspecies: Strain: AAV9 Pathovar: European Commission administrative Information Consent given by the Member State Competent Authority: Not known