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, multicentre, open label, single dose, dose-ranging clinical trial to investigate the safety and tolerability of a gene therapy vector rAAV2/5-PBGD for the treatment of Acute Intermittent Porphyria.

Proposed period of release:
01/08/2012 to 31/12/2013

Name of the Institute(s) or Company(ies)
Digna Biotech SL, Avda. Pio XII, 22 - Oficina 2
31008 Pamplona (Navarra);

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:
Genus: Dependovirus
Species: AAV-derived replication-deficient vector
The complete name of the selected recombinant vector is rAAV2/5.2-Ealb-hAAT-cohPBGDpolyA PBGD, also shortly named rAAV2/5-PBGD and coded as AMT-021 by the manufacturer.

Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
-DependovirusAdeno-associated Virus-Serotypes 2 and 5AAV2 and AAV5

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known