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 II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A

Proposed period of release:
01/07/2019 to 31/07/2023

Name of the Institute(s) or Company(ies)
Rocket Pharmaceuticals, Inc., ;

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

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

GMO characterization

GMO is a:
Other: mammals

Identity of the GMO:
Human hematopoietic progenitors CD34+ from Fanconi Anemia subtype A patients (Homo sapiens sapiens) transduced with self-inactivating (SIN) lentiviral vector PGK-FANCA.Wpre*.

Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known