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
An Open-Label Safety Study of Retinal Gene Therapy for Choroideremia with Bilateral, Sequential Administration of Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

Proposed period of release:
01/10/2017 to 31/12/2018

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

3. Is the same GMO release planned elsewhere in the Community?

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

If yes, notification number(s):
B/DE/15/PEI2422; B/DE/16/PEI2698;

GMO characterization

GMO is a:
DNA Virus

Identity of the GMO:
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 choroideremia.
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:
17/01/2018 00:00:00