Skip to main content

Table 2 The efficacy of CRISPR-based cancer therapeutics in several different clinical studies

From: Strategies to overcome the main challenges of the use of CRISPR/Cas9 as a replacement for cancer therapy

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 [81, 82]
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 [81,82,83]
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 B2 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 [84,85,86,87]
NCT02793856 China Sichuan University Metastatic Non-small Cell Lung Cancer 12 PD-1 KO T Cells Unspecified - Completed
(March 17, 2020)
[85,86,87,88,89,90,91,92]
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 [93,94,95,96,97,98]
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   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   Intellia Therapeutics Hereditary Transthyretin Amyloidosis 38 NTLA-2001 lipid nanoparticles Phase 1 March 2024 [102, 103]