Definition

A preparation of human allogeneic T lymphocytes gene-edited with the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 nuclease complex to disrupt expression of endogenous TCR and major histocompatibility complex (MHC) class I molecules and modified to express a chimeric antigen receptor (CAR) specific for the tumor-associated antigen (TAA) human cluster of differentiation 70 (CD70), with potential immunostimulating and antineoplastic activities. Upon introduction into the patient, the allogeneic CRISPR-Cas9 engineered anti-CD70 CAR-T cells CTX130 recognize and bind to CD70-overexpressing tumor cells. This may result in a specific cytotoxic T-lymphocyte (CTL)-mediated killing of CD70-positive tumor cells. CD70, the ligand for the costimulatory receptor CD27 and a member of the tumor necrosis factor (TNF) family, is found on the surfaces of various types of cancer cells. Disruption of endogenous TCR prevents graft-versus-host disease (GvHD); the disruption of MHC class I molecules increases the persistence of the CAR-T cells.