Table 1.
Current Ex vivo CRISPR-mediated cancer therapeutics in clinical trials
| Phase | Cancer Type | Delivery Mode | Editing Target | ID |
|---|---|---|---|---|
| I/II | B-cell leukemia | Electroporation of CRISPR sgRNA to disrupt endogenous TCR and B2M. | Allogeneic CD19-CAR modified T cells with βTCRα, TCRβ, β-2 microglobulin (B2M) knockout. | NCT03166878 |
| I/II | B cell leukemia, B cell lymphoma | Undefined | Allogeneic CD19 and CD20 or CD22 CAR-T cells. | NCT03398967 |
| I | Metastatic non-small cell lung cancer | Electroporation of Cas9 and sgRNA plasmids to target exon 2 of PD-1. | Modified autologous T cells with programmed cell death protein 1 (PD-1) knockout. | NCT02793856 |
| I | Esophageal cancer | Electroporation of CRISPR/Cas9 DNA to disrupt PD-1 | PD-1 knockout engineered T cells. | NCT03081715 |
| I | Advanced refractory cancer | Electroporation of Cas9 RNP with three sgRNA for gene editing of TRAC, TRBC, and PDCD1. | Knockdown of endogenous T cell receptor (TCR) and PD-1 and knock-in of cancer-specific TCR transgene in autologous isolated T-cells. | NCT03399448 |
| I | Solid tumors | Electroporation of Cas9 RNP with sgRNAs targeting PD-1 and TCR. | PD-1 and TCR gene knockout CAR-T Cells. | NCT03545815 |
| I | B-cell lymphoma, non-Hodgkin lymphoma | Undefined | Allogeneic CD19-directed CAR T cell immunotherapy (CTX110). | NCT04035434 |
| I | Relapsed or refractory acute leukemia lymphocytic | Electroporation of CRISPR sgRNA to disrupt endogenous HPK1 in Autologous T cells. | Autologous T Cells with endogenous hematopoietic progenitor kinase 1 (HPK1) knockout. | NCT04037566 |
| I | Multiple myeloma | Undefined | Anti-BCMA allogeneic engineered T cells (CTX120). | NCT04244656 |
| I | Gastro-intestinal (GI) cancer | Undefined | CISH (Cytokine-induced SH2 protein) modified tumor-infiltrating lymphocytes (TIL). | NCT04426669 |
| I | Relapsed or refractory T or B Cell lymphoma, renal cell carcinoma | Undefined | Anti-CD70 allogeneic engineered T cells (CTX130). |
NCT04438083
NCT04502446 |
| I | B acute lymphoblastic leukemia | Lentiviral vector CRISPR guides for genome editing of CD52 and TRAC and transient Cas9 protein. | Allogeneic engineered human T cells to disrupt CD52 and T cell receptor α (TRAC.) | NCT04557436 |
| I | Relapsed or refractory B cell non-Hodgkin lymphoma, various lymphoma | AAV vector with Cas9 chRDNA (CRISPR hybrid RNA-DNA) technology to insert CD19-specific CAR into T cell, knockout TRAC and PD-1. | Allogeneic anti-CD19 CAR-T cells (CB-010). | NCT04637763 |