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Notification report


Full notification file


General information

Notification Number
B/DE/18/PEI3354

Member State to which the notification was sent
Germany

Date of acknowledgement from the Member State Competent Authority
02/03/2018

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:
01/07/2018 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; Germany; Spain; France; United Kingdom; Italy; Netherlands; Sweden;

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

European Commission administrative information

Consent given by the Member State Competent Authority:
Yes
06/11/2018 00:00:00
Remarks: