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
Clinical study CTX001-121 titled: “A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects with Severe Sickle Cell Disease”

Proposed period of release:
01/10/2018 to 31/07/2022

Name of the Institute(s) or Company(ies)
CRISPR Therapeutics AG, Baarerstrasse 14
CH 6300 Zug, Switzerland;

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):

GMO characterization

GMO is a:
Other: mammal

Identity of the GMO:
The GMO/IMP (CTX001) consists of subject autologous peripheral blood-derived CD34+ human hematopoietic stem and progenitor cells (hHSPCs) modified ex vivo using the CRISPR-Cas9 gene editing technology. The CRISPR-Cas9 editing components are transiently introduced into the target cell population by electroporation as a ribonucleoprotein complex consisting of Cas9 and a gRNA that targets the erythroid lineage-specific enhancer of the BCL11A gene. This process does not use plasmid or viral vectors. The CRISPR-Cas9 gene editing technology is intended to disrupt the erythroid lineage-specific enhancer of the BCL11A gene on chromosome 2 in a site-specific and permanent fashion in order to reactivate transcription and expression of γ-globin, resulting in increased levels of fetal hemoglobin (HbF) for the treatment of sickle cell disease.
The cells will be used only for therapeutic purposes in the same subject from whom the cells were obtained (autologous application).

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:
06/02/2019 00:00:00