allogeneic CRISPR-Cas9 engineered anti-CD19 CAR T cells CTX112

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.