Notification report

Full notification file

General information

Notification Number

Member State to which the notification was sent

Date of acknowledgement from the Member State Competent Authority

Title of the Project
Phase I/II Gene Therapy Clinical Trial of scAAV9.U1a.SGSH for Mucopolysaccharidosis IIIA

Proposed period of release:
01/01/2016 to 30/09/2018

Name 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?

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

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.

scAAV9.U1a.SGSH 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 scAAV9.U1a.SGSH, as unique name for this recombinant vector.

Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
AAV2ParvoviridaeDependovirusDependovirusserotype 2-
AAV9ParvoviridaeDependovirusDependovirusSerotypes 9-

European Commission administrative information

Consent given by the Member State Competent Authority:
14/12/2015 00:00:00