Full notification fileGeneral informationNotification NumberB/ES/15/11Member State to which the notification was sentSpainDate of acknowledgement from the Member State Competent Authority10/11/2015Title of the ProjectPhase I/II Gene Therapy Clinical Trial of rAAV9.CMV.NAGLU for Mucopolysaccharidosis IIIBProposed period of release:01/01/2016 to 30/09/2018Name of the Institute(s) or Company(ies)Abeona Therapeutics, 10000 Cedar Avenue
Cleveland, OH 44106, USA;
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
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
European Commission administrative informationConsent given by the Member State Competent Authority:Yes14/12/2015 00:00:00Remarks: