Table 2 Gene editing clinical trials
From: From fiction to science: clinical potentials and regulatory considerations of gene editing
| Â | NCT (status) | Country/region | Sponsor | Disease | Target/modification | Nuclease | Delivery |
|---|---|---|---|---|---|---|---|
Ex vivo | NCT00842634 (completed) | US | Sangamo Therapeutics | HIV | CCR5 modified CD4+ T cells | ZFN | Adenoviral vector |
NCT01044654 (completed) | US | Sangamo Therapeutics | HIV | CCR5 modified CD4+ T cells | ZFN | Adenoviral vector | |
NCT01252641 (completed) | US | Sangamo Therapeutics | HIV | CCR5 modified CD4+ T cells | ZFN | Adenoviral vector | |
NCT02388594 (completed) | US | University of Pennsylvania | HIV | CCR5 modified CD34+ T cells | ZFN | mRNA electroporation | |
NCT02500849 (active, not recruiting) | US | City of Hope Medical Center | HIV | CCR5 modified CD34+ HSPCs | ZFN | mRNA electroporation | |
NCT03617198 (not yet recruiting) | US | University of Pennsylvania | HIV | CCR5 modified, C34-CXCR4, CD4 CAR T cells | ZFN | Not specified | |
NCT03666871 (not yet recruiting) | Not specified | Case Western Reserve University | HIV | CCR5 modified CD4+ T cells | ZFN | Not specified | |
NCT03190278 (recruiting) | US | Cellectis S.A. | Acute myeloid leukemia | Allogeneic CAR T cells targeting CD123, TCR disruption | TALEN | Not specified | |
NCT03164135 (recruiting) | China | Affiliated Hospital to Academy of Military Medical Sciences (China) | HIV-1 | CCR5 modified CD34+ HSPCs (from donor) | CRISPR/Cas | Not specified | |
NCT03655678 (recruiting) | Canada, Europe | Vertex Pharmaceuticals Incorporated | Beta Thalassemia | Autologous CD34+ HSPCs modified at the enhancer of the BCL11A gene | CRISPR/Cas9 | Ribonucleoprotein electroporation | |
NCT03728322 (not yet recruiting) | Not specified | Allife Medical Science and Technology Co., Ltd | Beta thalassemia | HBB gene correction in patient specific iHSCs | CRISPR/Cas9 | Not specified | |
NCT03745287 (recruiting) | US, Europe | Vertex Pharmaceuticals Incorporated | Sickle cell disease | Autologous CD34+ HSPCs modified at the enhancer of the BCL11A gene | CRISPR/Cas9 | Ribonucleoprotein electroporation | |
NCT03398967 (recruiting) | China | Chinese PLA General Hospital | B cell leukemia, B cell lymphoma | Allogeneic CD19 and CD20/22 CAR T cells | CRISPR/Cas9 | Not specified | |
NCT03166878 (recruiting) | China | Chinese PLA General Hospital | B cell leukemia, B cell lymphoma | Allogeneic CD19-directed CAR T cells; TCR and B2Â M disruption | CRISPR/Cas9 | Not specified | |
NCT03399448 (recruiting) | US | University of Pennsylvania | Multiple Myeloma, Melanoma, Synovial Sarcoma, Myxoid/Round Cell Liposarcoma | Autologous anti-NY-ESO CAR T cells, disruption of TCR and PD-1 | CRISPR/Cas | mRNA electroporation | |
NCT03690011 (not yet recruiting) | US | Baylor College of Medicine | T cell Acute Lymphoblastic Leukemia, T cell Acute Lymphoblastic Lymphoma, T-non-Hodgkin Lymphoma | Anti-CD7 CAR T cells, CD7 KO | CRISPR/Cas9 | Not specified | |
NCT03545815 (recruiting) | China | Chinese PLA General Hospital | Solid tumor | PD-1 and TCR KO anti-mesothelin CAR T cells | CRISPR/Cas9 | Not specified | |
NCT03747965 (recruiting) | China | Chinese PLA General Hospital | Solid tumor | Mesothelin-directed CAR T cells; PD-1 KO | CRISPR | Not specified | |
NCT03081715 (completed) | China | Hangzhou Cancer Hospital | Esophageal cancer | PD-1 KO T cells | CRISPR/Cas9 | Not specified | |
NCT02793856 (active, not recruiting) | China | Sichuan University | Metastatic non-small cell lung cancer | PD-1 KO T cells | CRISPR/Cas9 | Not specified | |
NCT03044743 (recruiting) | China | Yang Yang, Nanjing University Medical School | EBV positive advanced stage malignancies | PD-1 KO EBV-CTL cells | CRISPR/Cas9 | Not specified | |
NCT02863913 (withdrawn—no funding) | China | Peking University | Invasive Bladder Cancer Stage IV | PD-1 KO T cells | CRISPR/Cas9 | Not specified | |
NCT02867345 (withdrawn—no funding) | China | Peking University | Hormone Refractory Prostate Cancer | PD-1 KO T cells | CRISPR/Cas9 | Not specified | |
NCT02867332 (withdrawn—no funding) | China | Peking University | Metastatic renal cell carcinoma | PD-1 KO T cells | CRISPR/Cas9 | Not specified | |
In vivo | NCT03041324 (recruiting) | US | Sangamo Therapeutics | Mucopolysaccharidosis II | Insertion of corrected copy of Iduronate 2-Sulfatase gene into the Albumin locus | ZFN | AAV |
NCT02702115 (recruiting) | US | Sangamo Therapeutics | Mucopolysaccharidosis I | Insertion of corrected copy of α-l-iduronidase gene into the Albumin locus | ZFN | AAV | |
NCT02695160 (recruiting) | US, Europe | Sangamo Therapeutics | Hemophilia B | Insertion of a corrected copy of the factor 9 gene into the albumin locus | ZFN | AAV | |
NCT02800369 (active, not recruiting) | China | Huazhong University of Science and Technology | Human papillomavirus-Related malignant neoplasm | E7 | ZFN | Not specified | |
NCT03226470 (not yet recruiting) | China | Huazhong University of Science and Technology | Human papillomavirus-related malignant neoplasm | E6, E7 | TALEN | Plasmid in gel | |
NCT03057912 (unknown) | China | First Affiliated Hospital, Sun Yat-Sen University | Human papillomavirus-Related malignant neoplasm | E6, E7 | TALEN CRISPR/Cas9 | Plasmid in gel | |
NCT03872479 (not yet recruiting) | US | Allergan | Leber congenital amaurosis 10 | CEP290 | CRISPR/Cas9 | AAV |