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Notification report


Full notification file


General information

Notification Number
B/DE/17/PEI3143

Member State to which the notification was sent
Germany

Date of acknowledgement from the Member State Competent Authority
14/07/2017

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?
No

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

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:
Yes
17/01/2018 00:00:00
Remarks: