Table 1.
Current Clinical Trials Involving Gene Editing
| Title | Tool | Status | Country | Delivery | ID | Ref. |
|---|---|---|---|---|---|---|
| Cancer Immunotherapy | ||||||
| PD-1 knockout engineered T cells for advanced esophageal cancer | CRISPR-Cas9 | completed | China | ex vivo | NCT03081715 | 61 |
| PD-1 knockout engineered t cells for metastatic non-small cell lung cancer | CRISPR-Cas9 | active, not recruiting | China | ex vivo | NCT02793856 | 62 |
| Therapeutic vaccine plus PD-1 knockout in prostate cancer treatment | CRISPR-Cas9 | recruiting | China | ex vivo | NCT03525652 | 63 |
| PD-1 knockout EBV-CTLs for advanced stage Epstein-Barr virus (EBV) associated malignancies | CRISPR-Cas9 | recruiting | China | ex vivo | NCT03044743 | 64 |
| CD19 CAR and PD-1 knockout engineered T cells for CD19 positive malignant B cell derived leukemia and lymphoma | N.S. | not yet recruiting | China | ex vivo | NCT03298828 | 82 |
| Study of PD-1 gene-knocked out mesothelin-directed CAR-T cells with the conditioning of PC in mesothelin positive multiple solid tumors | CRISPR-Cas9 | recruiting | China | ex vivo | NCT03747965 | 83 |
| CAR T and PD-1 knockout engineered T cells for esophageal cancer | N.S. | recruiting | China | ex vivo | NCT03706326 | 84 |
| Anti-MUC1 CAR T cells and PD-1 knockout engineered T cells for NSCLC | N.S. | recruiting | China | ex vivo | NCT03525782 | 85 |
| CRISPR (HPK1) edited CD19-specific CAR-T cells (XYF19 CAR-T Cells) for CD19+ leukemia or lymphoma | CRISPR-Cas9 | recruiting | China | ex vivo | NCT04037566 | 86 |
| Study of UCART19 in pediatric patients with relapsed/refractory B acute lymphoblastic leukemia (PALL) | TALEN | active, not recruiting | US/EU/UK | ex vivo | NCT02808442 | 103 |
| Dose escalation study of UCART19 in adult patients with relapsed/refractory B cell acute lymphoblastic leukaemia (CALM) | TALEN | active, not recruiting | US/EU/UK/Japan | ex vivo | NCT02746952 | 104 |
| Safety and efficacy of ALLO-501 anti-CD19 allogeneic CAR T cells in adults with relapsed/refractory large B cell or follicular lymphoma (ALPHA) | TALEN | recruiting | US | ex vivo | NCT03939026 | 105 |
| Safety and efficacy of ALLO-715 BCMA allogenic CAR T cells in in adults with relapsed or refractory multiple myeloma (UNIVERSAL) | TALEN | recruiting | US | ex vivo | NCT04093596 | 106 |
| A study to evaluate the long-term safety of patients with advanced lymphoid malignancies who have been previously administered with UCART19/ALLO-501 | TALEN | enrolling by invitation | US/EU/UK/Japan | ex vivo | NCT02735083 | 107 |
| A study evaluating UCART019 in patients with relapsed or refractory CD19+ leukemia and lymphoma | CRISPR-Cas9 | recruiting | China | ex vivo | NCT03166878 | 112 |
| A safety and efficacy study evaluating CTX110 in subjects with relapsed or refractory B cell malignancies | CRISPR-Cas9 | recruiting | US/Australia/Germany | ex vivo | NCT04035434 | 115 |
| A safety and efficacy study evaluating CTX120 in subjects with relapsed or refractory multiple myeloma | CRISPR-Cas9 | recruiting | US/Australia | ex vivo | NCT04244656 | 116 |
| CTA101 UCAR-T cell injection for treatment of relapsed or refractory CD19+ B cell acute lymphoblastic leukemia | CRISPR-Cas9 | recruiting | China | ex vivo | NCT04154709 | 117 |
| Phase I study of UCART22 in patients with relapsed or refractory CD22+ B cell acute lymphoblastic leukemia (BALLI-01) | TALEN | recruiting | US | ex vivo | NCT04150497 | 118 |
| CTA101 in the treatment of relapsed or refractory diffuse large B cell lymphoma | CRISPR-Cas9 | not yet recruiting | China | ex vivo | NCT04026100 | 119 |
| A feasibility and safety study of universal dual specificity CD19 and CD20 or CD22 CAR-T cell immunotherapy for relapsed or refractory leukemia and lymphoma | CRISPR-Cas9 | recruiting | China | ex vivo | NCT03398967 | 120 |
| Study evaluating safety and efficacy of UCART123 in patients with acute myeloid leukemia (AMELI-01) | TALEN | recruiting | US | ex vivo | NCT03190278 | 121 |
| Study evaluating safety and efficacy of UCART targeting CS1 in patients with relapsed/refractory multiple myeloma (MELANI-01) | TALEN | recruiting | US | ex vivo | NCT04142619 | 122 |
| Anti-CD19 U-CAR-T cell therapy for B cell hematologic malignancies | N.S. | not yet recruiting | China | ex vivo | NCT04264039 | 123 |
| Anti-CD7 U-CAR-T cell therapy for T/NK cell hematologic malignancies | N.S. | not yet recruiting | China | ex vivo | NCT04264078 | 124 |
| Efficacy and safety evaluation of BCMA-UCART | N.S. | recruiting | China | ex vivo | NCT03752541 | 125 |
| Safety and efficacy evaluation of CD19-UCART | N.S. | recruiting | China | ex vivo | NCT03229876 | 126 |
| The clinical study of CD19 UCAR-T cells in patients with B cell acute lymphoblastic leukemia (B-ALL) | N.S. | recruiting | China | ex vivo | NCT04166838 | 127 |
| NY-ESO-1-redirected CRISPR (TCRendo and PD1) edited t cells (NYCE T cells) | CRISPR-Cas9 | terminated | US | ex vivo | NCT03399448 | 133 |
| Study of CRISPR-Cas9 mediated PD-1 and TCR gene-knocked out mesothelin-directed CAR-T cells in patients with mesothelin positive multiple solid tumors | CRISPR-Cas9 | recruiting | China | ex vivo | NCT03545815 | 134 |
| Cell therapy for high risk T cell malignancies using CD7-specific CAR expressed on autologous T cells | CRISPR-Cas9 | not yet recruiting | US | ex vivo | NCT03690011 | 144 |
| Cervical Cancer | ||||||
| Study of molecular-targeted therapy using zinc finger nuclease in cervical precancerous lesions | ZFN | N.S. | China | in vivo | NCT02800369 | 160 |
| Study of targeted therapy using transcription activator-like effector nucleases in cervical precancerous lesions | TALEN | N.S. | China | in vivo | NCT03226470 | 161 |
| A safety and efficacy study of TALEN and CRISPR/Cas9 in the treatment of HPV-related cervical intraepithelial neoplasia | CRISPR-Cas9 TALEN |
N.S. | China | in vivo | NCT03057912 | 162 |
| HIV Infection and AIDS | ||||||
| Autologous T cells genetically modified at the CCR5 gene by zinc finger nucleases SB-728 for HIV | ZFN | completed | US | ex vivo | NCT00842634 | 189 |
| Phase 1 dose escalation study of autologous t cells genetically modified at the CCR5 gene by zinc finger nucleases in HIV-infected patients | ZFN | completed | US | ex vivo | NCT01044654 | 190 |
| Repeat doses of SB-728mR-T after cyclophosphamide conditioning in HIV-infected subjects on HAART | ZFN | completed | US | ex vivo | NCT02225665 | 191 |
| A phase I study of T cells genetically modified at the CCR5 gene by zinc finger nucleases SB-728mR in HIV-infected patients | ZFN | completed | US | ex vivo | NCT02388594 | 192 |
| Dose escalation study of cyclophosphamide in HIV-infected subjects on HAART receiving SB-728-T | ZFN | completed | US | ex vivo | NCT01543152 | 193 |
| CCR5-modified CD4+ T cells for HIV infection (TRAILBLAZER) | ZFN | recruiting | US | ex vivo | NCT03666871 | 194 |
| Study of autologous T cells genetically modified at the CCR5 gene by zinc finger nucleases in HIV-infected subjects | ZFN | completed | US | ex vivo | NCT01252641 | 195 |
| Long-term follow-up of HIV subjects exposed to SB-728-T or SB-728mR-T | ZFN | enrolling by invitation | US | ex vivo | NCT04201782 | 197 |
| Safety study of zinc finger nuclease CCR5-modified hematopoietic stem/progenitor cells in HIV-1 infected patients | ZFN | active, not recruiting | US | ex vivo | NCT02500849 | 203 |
| Safety of transplantation of CRISPR CCR5 modified CD34+ cells in HIV-infected subjects with hematological malignances | CRISPR-Cas9 | recruiting | China | ex vivo | NCT03164135 | 204 |
| CD4 CAR+ ZFN-modified T cells in HIV therapy | ZFN | active, not recruiting | US | ex vivo | NCT03617198 | 206 |
| β-thalassemia and Sickle Cell Disease | ||||||
| A safety and efficacy study evaluating CTX001 in subjects with transfusion-dependent β-thalassemia | CRISPR-Cas9 | recruiting | US/Canada/EU/UK | ex vivo | NCT03655678 | 263 |
| A study to assess the safety, tolerability, and efficacy of ST-400 for treatment of transfusion-dependent beta-thalassemia (TDT) | ZFN | active, not recruiting | US | ex vivo | NCT03432364 | 264 |
| A safety and efficacy study evaluating CTX001 in subjects with severe sickle cell disease | CRISPR-Cas9 | recruiting | US/Canada/EU | ex vivo | NCT03745287 | 265 |
| A study to assess the safety, tolerability, and efficacy of BIVV003 for autologous hematopoietic stem cell transplantation in patients with severe sickle cell disease (BIVV003) | ZFN | recruiting | US | ex vivo | NCT03653247 | 266 |
| A long-term follow-up study in subjects who received CTX001 | CRISPR-Cas9 | enrolling by invitation | US/EU | ex vivo | NCT04208529 | 267 |
| iHSCs with the gene correction of HBB intervent subjests with β-thalassemia mutations | CRISPR-Cas9 | not yet recruiting | N.S. | ex vivo | NCT03728322 | 280 |
| Hemophilia | ||||||
| Ascending dose study of genome editing by zinc finger nuclease therapeutic SB-FIX in subjects with severe hemophilia B | ZFN | active, not recruiting | US | in vivo | NCT02695160 | 289 |
| Mucopolysaccharidoses | ||||||
| Ascending dose study of genome editing by the zinc finger nuclease (ZFN) therapeutic SB-318 in subjects with MPS I | ZFN | active, not recruiting | US | in vivo | NCT02702115 | 319 |
| Ascending dose study of genome editing by the zinc finger nuclease (ZFN) therapeutic SB-913 in subjects with MPS II | ZFN | active, not recruiting | US | in vivo | NCT03041324 | 320 |
| Leber’s Congenital Amaurosis | ||||||
| Single ascending dose study in participants with LCA10 | CRISPR-Cas9 | recruiting | US | in vivo | NCT03872479 | 329 |
N.S., not specified.