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Notification report


Full notification file


General information

Notification Number
B/DE/19/PEI3590

Member State to which the notification was sent
Germany

Date of acknowledgement from the Member State Competent Authority
29/10/2018

Title of the Project
Phase I/II gene transfer clinical trial of rAAV9.CMV.hNAGLU for Mucopolysaccharidosis (MPS) IIIB

Proposed period of release:
01/01/2019 to 30/09/2021

Name of the Institute(s) or Company(ies)
Abeona Therapeutics Europe SL., Avenida de Manoteras 30, A207-208
28050 Madrid, Spain;


3. Is the same GMO release planned elsewhere in the Community?
Yes:
Spain; France; United Kingdom;

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:
This is a recombinant AAV vector (replication deficient) derived from serotype 9 also named rh14.

rAAV9.CMV.NAGLU is a genetically modified organism derived from wild type adeno- associated virus serotype 9 (rh14). This adeno-associated virus (AAV) is a member of the genus Dependovirus, which lies within the Parvoviridae family. Serotype 9 (also named rh14) was isolated from mammals, specifically humans and was serologically distinct from the known serotypes (for more information about AAV serotypes origin see Gao et. al. 2004 reference).
The identity of the genetically modified organism is rAAV9.CMV.NAGLU, as unique name for this recombinant vector.


Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
AAV2 pseudotyped with capsids of AAV1ParvoviridaeDependovirusDependovirusserotype 2-
AAV9ParvoviridaeDependovirusDependovirusSerotype 9-

European Commission administrative information

Consent given by the Member State Competent Authority:
Yes
24/06/2019 00:00:00
Remarks: