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Notification report


Full notification file


General information

Notification Number
B/ES/19/26

Member State to which the notification was sent
Spain

Date of acknowledgement from the Member State Competent Authority
25/11/2019

Title of the Project
Clinical study CRSP-ONC-002 titled: “A Phase 1Dose Escalation and Cohort Expansion Study of Safety and Efficacy of Anti-BCMA Allogeneic CRISPR-Cas9–Engineered T Cells (CTX120) in Subjects with Relapsed or Refractory Multiple Myeloma”

Proposed period of release:
01/03/2020 to 30/11/2026

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?
Yes:
Germany; United Kingdom; Sweden;

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

GMO characterization

GMO is a:
Other: mammal

Identity of the GMO:
Genus: Homo; Species: H.Sapiens (T cells genetically modified)

The GMO (CTX120) consists of human allogeneic T cells that are genetically modified ex vivo using CRISPR/Cas9 genomic editing components and a recombinant adeno-associated viral (rAAV) vector. The drug product is prepared from healthy donor peripheral blood mononuclear cells obtained via a standard leukapheresis procedure. The mononuclear cells are enriched for T cells and activated with anti-CD3-CD28 antibody-coated beads, then electroporated with CRISPR/Cas9 ribonucleoprotein complexes consisting of Cas9 and single guide RNA and transduced with a chimeric antigen receptor (CAR) gene-containing rAAV vector. The modified T cells are expanded in cell culture, purified, formulated into a suspension, and cryopreserved. The CRISPR/Cas9 system, along with an AAV-derived donor DNA template, has been used to create three edits in the genome of healthy donor T cells: T cell receptor alpha constant region (TRAC) disruption, β2-microglobulin (B2M) disruption, and site-specific incorporation of DNA sequence encoding a BCMA-targeting CAR into the TRAC locus. The gene edits are intended to reduce the probability of graft versus host disease (GvHD), improve persistence by reducing the probability of host rejection and direct the modified T cells towards BCMA expressing tumor cells.
The cells will be used only for therapeutic purposes.


Information relating to the recipient or parental organisms from wich the GMO is derived
Common NameGenusSpeciesSubspeciesStrainPathovar
HumanHomoSapiens-T cells-

European Commission administrative information

Consent given by the Member State Competent Authority:
Not known