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Notification report


Full notification file


General information

Notification Number
B/ES/18/07

Member State to which the notification was sent
Spain

Date of acknowledgement from the Member State Competent Authority
19/06/2018

Title of the Project
Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous Infusion.

Proposed period of release:
16/07/2018 to 31/12/2021

Name of the Institute(s) or Company(ies)
AveXis, Inc., 2275 Half Day Road, Suite 200 Bannockburn, IL 60015, United States;


3. Is the same GMO release planned elsewhere in the Community?
Yes:
Belgium; Spain; France; United Kingdom; Italy; Netherlands; Sweden; Czech Republic;

Has the same GMO been notified elsewhere by the same notifier?
Yes

If yes, notification number(s):


GMO characterization

GMO is a:
DNA Virus

Identity of the GMO:
Family: Parvoviridae
Genus: Dependovirus
Species: Adeno-associated Virus (AAV9-derived replication-deficient viral vector containing human SMN cDNA)


Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
AAV2/9DependovirusAdeno‚ÄźAssociated Virus--Serotype 2 and 9

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known