Table 2 The efficacy of CRISPR-based cancer therapeutics in several different clinical studies
Clinical Trials Identification | Country | Developer | Disease | Number of participants | Target Gene/modification | Delivery | Study Phase | Estimated Study Completion Date | References |
|---|---|---|---|---|---|---|---|---|---|
NCT03655678 | Canada, Europe | Vertex Pharmaceuticals Incorporated | Beta Thalassemia | 45 | Analogous CD34+ HSPCs that have been reprogrammed at the BCL11A gene enhancer. | Ribonucleoprotein electroporation | Phase 2 Phase 3 | August 2024 | |
NCT03728322 | Unknown | Allife Medical Science and Technology Co., Ltd. | Beta Thalassemia | 12 | HBB gene is corrected in iHSCs patient-specific | Unspecified | Early Phase 1 | January 31, 2021 | - |
NCT04205435 | China | Bioray Laboratories | Beta Thalassemia Major | 12 | Autologous hematopoietic stem cells gene-edited with β-globin restoration. | Unspecified | Phase 1 Phase 2 | December 1, 2023 | [81] |
NCT04211480 | China | Bioray Laboratories | Thalassemia Major | 12 | Gene-edited autologous hematopoietic stem cells with γ-globin expression | Unspecified | / | June 1, 2023 | - |
NCT03745287 | US, Europe | Vertex Pharmaceuticals Incorporated | Severe Sickle cell disease | 45 | Autologous CD34+ HSPCs modified at the enhancer of the BCL11A gene | Ribonucleoprotein electroporation | Phase 1 Phase 2 | October 2024 | |
NCT04774536 | US | Mark Walters, MD | Sickle cell disease | 9 | Autologous CD34+ HSPCs modification | Ribonucleoprotein | Phase 1 Phase 2 | December 1, 2026 | - |
NCT04037566 | China | Xijing Hospital | 1. Leukemia Lymphocytic Acute (All) Refractory 2. Lymphoma, B-Cell | 40 | CRISPR gene-edited to eliminate endogenous HPK1(XYF19 CAR-T cell) | Ribonucleoprotein electroporation | Phase 1 | August 2024 | - |
NCT03398967 | China | Chinese PLA General Hospital | (1) B cell leukemia, (2) B cell lymphoma | 80 | Allogeneic CD11 and CD20/22 directed CAR T cells | Unspecified | Phase1 Phase2 | May 20, 2022 | - |
NCT03166878 | China | Chinese PLA General Hospital | (1) B cell leukemia, (2) B cell lymphoma | 80 | Allogeneic CD19-directed CAR T cells; TCR and B2Â M disruption | RNA electroporation | Phase1 Phase2 | May 21, 2022 | - |
NCT03690011 | US | Baylor College of Medicine | 1. T-cell Acute Lymphoblastic Leukemia 2. T-cell Acute Lymphoblastic Lymphoma 3. T-non-Hodgkin Lymphoma | 21 | Anti-CD7 CAR T cells, CD7 KO | Unspecified | Phase1 | May1, 2038 | - |
NCT04637763 | US | Caribou Biosciences, Inc | 1. Lymphoma, Non-Hodgkin 2. Relapsed Non-Hodgkin Lymphoma 3. Refractory B-Cell Non-Hodgkin Lymphoma 4. Non-Hodgkin Lymphoma 5. Lymphoma 6. B Cell Lymphoma 7. B Cell Non-Hodgkin’s Lymphoma | 50 | CRISPR-edited allogeneic CAR-T cell therapy- CRISPR-edited targeting CD19 (CB-010) | Unspecified | Phase 1 | September 2025 | - |
NCT03545815 | China | Chinese PLA General Hospital | Solid Tumor, Adult | 10 | PD-1 and TCR KO anti-mesothelin CAR T cells | Unspecified | Phase 1 | December 30, 2020 | - |
NCT03747965 | China | Chinese PLA General Hospital | Solid Tumor, Adult | 10 | Mesothelin-directed CAR-T cells | Unspecified | Phase 1 | May 2020 | - |
NCT03081715 | China | Hangzhou Cancer Hospital | Advanced Esophageal Cancer | 16 | PD-1 KO T Cells | Unspecified | - | Completed | |
NCT02793856 | China | Sichuan University | Metastatic Non-small Cell Lung Cancer | 12 | PD-1 KO T Cells | Unspecified | - | Completed (March 17, 2020) | |
NCT03044743 | China | Yang Yang, Nanjing University Medical School | 1. Stage IV Gastric Carcinoma 2. Stage IV Nasopharyngeal Carcinoma 3. T-Cell Lymphoma Stage IV 4. Stage IV Adult Hodgkin Lymphoma 5. Stage IV Diffuse Large B-Cell Lymphoma | 20 | PD Knockout EBV-CTL | Unspecified | Phase 1 Phase 2 | March 2022 | |
NCT04426669 | US, UK | Intima Bioscience, Inc | Gastrointestinal cancers | 20 | Gene Encoding autologous CISH-inactivated TILs | Unspecified | Phase 1 Phase 2 | October 2022 | - |
NCT03872479 | US | Editas Medicine, Inc. | 1. Blindness 2. Leber Congenital Amaurosis 10 3. Eye Diseases | 18 | Eliminate the mutation on the CEP290 gene | AAV | Phase 1 Phase 2 | March 22, 2024 | [99] |
NCT04560790 | China | Shanghai BDgene Co., Ltd | 1. Viral Keratitis 2. Blindness Eye 3. Herpes Simplex Virus Infection 4. Cornea | 6 | BD11 CRISPR/Cas9 mRNA Instantaneous Gene Editing | Unspecified | Phase 1 Phase 2 | May 2022 | - |
NCT03332030 | US | Roger Packer | 1. Neurofibromatosis Type 1 2. Tumors of the Central Nervous System | 20 | Fix NF1 mutation allele | Unspecified | - | July 1, 2025 | - |
NCT03164135 | China | Affiliated Hospital to Academy of Military Medical Sciences | HIV-1-infection | 5 | CD34+ hematopoietic stem/progenitor cells From donor are treated with CRISPR/Cas9 targeting CCR5 gene | Unspecified | Not Applicable | May 20, 2021 | [100] |
NCT04244656 | Swiss/American | CRISPR Therapeutics AG | Multiple Myeloma | 80 | CTX120 B-cell maturation antigen (BCMA)-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo | Unspecified | Phase1 | January 2027 | [101] |
NCT04035434 | Swiss/American | CRISPR Therapeutics AG | 1. B-cell Malignancy 2. Non-Hodgkin Lymphoma 3. B-cell Lymphoma 4. Adult B Cell ALL | 143 | CTX110 (CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo | Unspecified | Phase1 | August 2026 | - |
NCT04767308 | China | Huazhong University of Science and Technology | 1. CD5+ Relapsed/Refractory hematopoietic malignancies 2. Chronic lymphocytic leukemia (CLL) 3. Mantle Cell Lymphoma (MCL) 4. Diffuse large B-cell lymphoma (DLBCL) 5. Follicular lymphoma (FL) 6. Peripheral T-cell lymphomas (PTCL) | 18 | CT125A chimeric antigen receptor (CAR) T cells | Unspecified | Early phase 1 | December 2023 | - |
NCT04417764 | China | Central South University | Advanced Hepatocellular Carcinoma | 10 | PD-1 KO T Cells | Unspecified | Phase 1 | December 31, 2021 | - |
NCT03525652 | Â | The First Affiliated Hospital of Guangdong Pharmaceutical University | Prostate Cancer | 30 | PD-1 KO T Cells | Unspecified | Phase 1 Phase 2 | August 30, 2021 | - |
NCT04601051 | Â | Intellia Therapeutics | Hereditary Transthyretin Amyloidosis | 38 | NTLA-2001 | lipid nanoparticles | Phase 1 | March 2024 |