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Notification report


Full notification file


General information

Notification Number
B/ES/20/05

Member State to which the notification was sent
Spain

Date of acknowledgement from the Member State Competent Authority
12/02/2020

Title of the Project
A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria and Plasma Phe Levels > 600 ╬╝mol/L

Proposed period of release:
01/03/2020 to 30/03/2025

Name of the Institute(s) or Company(ies)
BioMarin Pharmaceutical Inc., ;


3. Is the same GMO release planned elsewhere in the Community?
Yes:
Germany; United Kingdom; Italy;

Has the same GMO been notified elsewhere by the same notifier?
No

GMO characterization

GMO is a:
DNA Virus

Identity of the GMO:
Genus: Dependoparvovirus,
Species: Adeno-associated virus/ serotype 5 (AAV 5)


Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
AAVDependoparvovirusAdeno-associated VirusSerotype 5--

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known