drug_type
RELEVANT_DRUG
intervention_type
Biological (cell-based immunotherapy; CAR T-cell therapy)
drug_description
An allogeneic, CD19-directed CAR T-cell therapy engineered ex vivo using CRISPR-Cas9; administered as a single IV infusion after lymphodepleting chemotherapy. Donor T cells are modified to express a CD19-specific chimeric antigen receptor that activates cytotoxic pathways (perforin/granzyme release and cytokine-mediated killing) to lyse malignant B cells in relapsed/refractory B-cell malignancies.
nci_thesaurus_concept_id
C199293
nci_thesaurus_preferred_term
Allogeneic CRISPR-Cas9 Engineered Anti-CD19 CAR T-cells CTX112
nci_thesaurus_definition
A preparation of human allogeneic T-lymphocytes transduced with a chimeric antigen receptor (CAR) specific for the tumor-associated antigen (TAA) CD19 and gene-edited with the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 nuclease complex to eliminate endogenous TCR, transforming growth factor-beta receptor II (TGFbRII), and Regnase-1, with potential immunostimulating and antineoplastic activities. Upon introduction into the patient, the allogeneic CRISPR-Cas9 engineered anti-CD19 CAR T-cells CTX112 recognize and bind to CD19-expressing tumor cells. This may result in a specific cytotoxic T-lymphocyte (CTL)-mediated killing of CD19-positive tumor cells. Removal of endogenous TCR reduces the risk of graft-versus-host disease (GvHD). CD19 antigen is a B-cell specific cell surface antigen expressed in all B-cell lineage malignancies. Incorporation of the Regnase-1 and TGFBR2 double knockout increases the potency, persistence and efficacy of the CAR-T cells and enhances anti-tumor activity.
drug_mesh_term
Chimeric Antigen Receptor T-Cell Immunotherapy
drug_category
CAR T
drug_class
Cellular Therapy
drug_delivery_route
Intravenous
drug_mechanism_of_action
Allogeneic donor T cells are CRISPR-Cas9–engineered to express a CD19-specific chimeric antigen receptor and to knock out endogenous TCR, TGFBR2, and Regnase-1. After infusion, CAR binding to CD19 on B cells activates cytotoxic pathways (perforin/granzyme release and cytokine-mediated killing) to eliminate CD19-positive malignant B cells, while edits reduce GVHD risk and enhance potency and persistence.
drug_name
CTX112
nct_id_drug_ref
NCT05643742