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
A Phase 3 Clinical Trial Of Retinal Gene Therapy For Choroideremia
Using An Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

Proposed period of release:
01/07/2016 to 30/11/2017

Name of the Institute(s) or Company(ies)
NightstaRx Ltd, ;

3. Is the same GMO release planned elsewhere in the Community?
Denmark; Finland; United Kingdom; Netherlands;

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

GMO characterization

GMO is a:
DNA Virus

Identity of the GMO:
Identity of the GMO (genus and species)
AAV2-REP1 is an adeno-associated virus-2 (AAV2) based gene therapy vector. The vector
genome is comprised of an expression cassettewith the human cDNA encoding REP1. The
cDNA fragment was originally isolated from a human retinal cDNA library from unaffected
individuals. AAV2-REP1 is a potential gene therapy medicinal product for the treatment of

The parental virus concerned in this summary is a primate (human) adeno-associated virus (AAV)
with the following taxonomy:
Group: Group II (ss DNA)
Family: Parvoviridae
Genus: Dependovirus
Species: Adeno-associated virus

Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
AAV2DependovirusAdeno-associated Virus--AAV2

European Commission administrative information

Consent given by the Member State Competent Authority:
31/10/2016 00:00:00