Table 1.
Ongoing clinical trials of CRISPR/Cas9 based gene therapies.
All clinical trials listed in this table are based on ClinicalTrials.gov.117 AAVS, adeno-associated viruses; hHSPCs, human hematopoietic stem and progenitor cells; EBV-CTLs, Epstein-Barr virus-specific cytotoxic T lymphocytes; CAR-T, chimeric antigen receptor (CAR) T-cell; iHSCs, induced hematopoietic stem cells; MSCs, mesenchymal stem cells; CISH, cytokine-induced SH2 protein; TIL, tumor infiltrating lymphocytes.
| Study Title | Conditions/Disease | Target Gene | Type | Delivery Systems | Phase | Status | ClinicalTrials.gov identifier |
|---|---|---|---|---|---|---|---|
| Single Ascending Dose Study in Participants With LCA10 | Blindness; Leber Congenital Amaurosis 10 Vision Disorders Eye Diseases Retinal Disease | CEP290 (Correction) | Virus (AAV5) | Adeno-associated virus | Phase 1/2 | Recruiting | NCT03872479 |
| A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia | Beta-Thalassemia Thalassemia Genetic Diseases, Inborn Hematologic Diseases Hemoglobinopathies | BCL11A | CD34+ hHSPCs | Ex vivo | Phase 1/2 | Recruiting | NCT03655678 |
| A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease | Sickle Cell Disease Hematological Diseases Hemoglobinopathies | BCL11A | CD34+ hHSPCs | Ex vivo | Phase 1/2 | Recruiting | NCT03745287 |
| PD-1 Knockout EBV-CTLs for Advanced Stage Epstein-Barr Virus (EBV) Associated Malignancies | Stage IV Gastric Carcinoma Stage IV Nasopharyngeal Carcinoma T-Cell Lymphoma Stage IV Stage IV Adult Hodgkin Lymphoma Stage IV Diffuse Large B-Cell Lymphoma | PD-1 | EBV-CTLs | Ex vivo | Phase 1/2 | Recruiting | NCT03044743 |
| A Study of Metastatic Gastrointestinal Cancers Treated With Tumor Infiltrating Lymphocytes in Which the Gene Encoding the Intracellular Immune Checkpoint CISH Is Inhibited Using CRISPR Genetic Engineering | Gastrointestinal Epithelial Cancer Gastrointestinal Neoplasms Cancer of Gastrointestinal Tract Cancer, Gastrointestinal Gastrointestinal Cancer Colo-rectal Cancer Pancreatic Cancer Gall Bladder Cancer Colon Cancer Esophageal Cancer Stomach Cancer | CISH | TIL | Ex vivo | Phase 1/2 | Recruiting | NCT04426669 |
| A Safety and Efficacy Study Evaluating CTX110 in Subjects With Relapsed or Refractory B-Cell Malignancies | B-cell Malignancy Non-Hodgkin Lymphoma B-cell Lymphoma | CD19 | CAR-T | Ex vivo | Phase 1/2 | Recruiting | NCT04035434 |
| 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 | B Cell Leukemia B Cell Lymphoma | CD19 and CD20 or CD22 | CAR-T | Ex vivo | Phase 1/2 | Recruiting | NCT03398967 |
| A Study Evaluating UCART019 in Patients With Relapsed or Refractory CD19+ Leukemia and Lymphoma | B Cell Leukemia B Cell Lymphoma | CD19 | CAR-T | Ex vivo | Phase 1/2 | Recruiting | NCT03166878 |
| A Safety and Efficacy Study Evaluating CTX120 in Subjects With Relapsed or Refractory Multiple Myeloma | Multiple Myeloma | BCMA | CAR-T | Ex vivo | Phase 1 | Recruiting | NCT04244656 |
| Study of PD-1 Gene-knocked Out Mesothelin-directed CAR-T Cells With the Conditioning of PC in Mesothelin Positive Multiple Solid Tumors | Solid Tumor, Adult | PD-1 | CAR-T | Ex vivo | Phase 1 | Recruiting | NCT03747965 |
| Cell Therapy for High Risk T-Cell Malignancies Using CD7-Specific CAR Expressed On Autologous T Cells | T-cell Acute Lymphoblastic Leukemia T-cell Acute Lymphoblastic Lymphoma T-non-Hodgkin Lymphoma | CD-7 | CAR-T | Ex vivo | Phase 1 | Not yet recruiting | NCT03690011 |
| 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. | Solid Tumor, Adult | PD-1/TCR | CAR-T | Ex vivo | Phase 1 | Recruiting | NCT03545815 |
| CRISPR (HPK1) Edited CD19-specific CAR-T Cells (XYF19 CAR-T Cells) for CD 19+ Leukemia or Lymphoma | Leukemia Lymphocytic Acute (ALL) in Relapse Leukemia Lymphocytic Acute (All) Refractory Lymphoma, B-Cell CD 19 Positive | CD19 | CAR-T | Ex vivo | Phase 1 | Recruiting | NCT04037566 |
| A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory Renal Cell Carcinoma | Renal Cell Carcinoma | CD70 | CAR-T | Ex vivo | Phase 1 | Recruiting | NCT04438083 |
| TACE Combined With PD-1 Knockout Engineered T Cell in Advanced Hepatocellular Carcinoma | Advanced Hepatocellular Carcinoma | PD-1 | T cells | Ex vivo | Phase 1 | Recruiting | NCT04417764 |
| PD-1 Knockout Engineered T Cells for Metastatic Non-small Cell Lung Cancer | Metastatic Non-small Cell Lung Cancer | PD-1 | T cells | Ex vivo | Phase 1 | Active, not recruiting | NCT02793856 |
| A Safety and Efficacy Study of TALEN and CRISPR/Cas9 in the Treatment of HPV-related Cervical Intraepithelial Neoplasial | Human Papillomavirus-Related Malignant Neoplasm | E6/E7 | Plasmid | Gel | Phase 1 | Unknown | NCT03057912 |
| Cell Therapy for High Risk T-Cell Malignancies Using CD7-Specific CAR Expressed On Autologous T Cells | T-cell Acute Lymphoblastic Leukemia T-cell Acute Lymphoblastic Lymphoma T-non-Hodgkin Lymphoma | CD7 | CAR-T | Ex vivo | Phase 1 | Not yet recruiting | NCT03690011 |
| iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations | Thalassemia | HBB (Correction) | iHSCs | Ex vivo | Early Phase 1 | Not yet recruiting | NCT03728322 |