Table 2.
Currently active clinical trials based on CRISPR/Cas technology.
| NCT Number | Title | Status | Conditions | Interventions | Phase | Enrollment | Sponsor/Collaborators |
|---|---|---|---|---|---|---|---|
| NCT04774536 | Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease |
Not yet Recruiting |
Sickle Cell Disease | CRISPR_SCD001 | I/II | 9 | Mark Walters, MD; University of California, Los Angeles; University of California, Berkeley; University of California, San Francisco |
| NCT03057912 | A Safety and Efficacy Study of TALEN and CRISPR/Cas9 in the Treatment of HPV-related Cervical Intraepithelial Neoplasia# |
Unknown Status |
Human Papillomavirus- Related Malignant Neoplasm |
CRISPR/Cas9 | I | 60 | First Affiliated Hospital, Sun Yat- Sen University; Jingchu University of Technology |
| NCT04426669 | 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 |
Recruiting | Gastrointestinal Epithelial Cancer; Gastrointestinal Neoplasms; Cancer of Gastrointestinal Tract; Gastrointestinal Cancer; Colo-rectal Cancer; Pancreatic Cancer; Gall Bladder Cancer; Colon Cancer; Esophageal Cancer; Stomach Cancer |
Tumor-Infiltrating Lymphocytes (TIL) |
I/II | 20 | Intima Bioscience, Inc.; Masonic Cancer Center, University of Minnesota |
| NCT03164135 | Safety of Transplantation of CRISPR CCR5 Modified CD34+ Cells in HIVinfected Subjects With Hematological Malignances |
Unknown Status |
HIV-1-infection | CCR5 gene modification | N/A | 5 | Affiliated Hospital to Academy of Military Medical Sciences; Peking University; Capital Medical University |
| NCT04560790 | Safety and Efficacy of CRISPR/Cas9 mRNA Instantaneous Gene Editing Therapy to Treat Refractory Viral Keratitis |
Completed | Viral Keratitis; Blindness Eye; Herpes Simplex Virus Infection; Cornea |
BD111 Adult single group Dose |
N/A | 3 | Shanghai BDgene Co., Ltd.; Eye & ENT Hospital of Fudan University |
| NCT04990557 | CRISPR/Cas9-modified Human T Cell ( PD-1and ACE2 Knockout Engineered T Cells ) for Inducing Longterm Immunity in COVID-19 Patients |
Not yet Recruiting |
COVID-19 Respiratory Infection |
PD-1 and ACE2 Knockout T Cells |
I/II | 16 | Mahmoud Ramadan mohamed Elkazzaz; Kafrelsheikh University |
| NCT03545815 | 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. |
Unknown Status |
Solid Tumor, Adult | anti-mesothelin CAR-T cells | I | 10 | Chinese PLA General Hospital |
| NCT04037566 | CRISPR (HPK1) Edited CD19-specific CAR-T Cells (XYF19 CAR-T Cells) for CD19+ Leukemia or Lymphoma. |
Recruiting | Leukemia Lymphocytic Acute (ALL) in Relapse; Leukemia Lymphocytic Acute (All) Refractory |
XYF19 CAR-T cell | I | 40 | Xijing Hospital; Xi'An Yufan Biotechnology Co.,Ltd |
| NCT05566223 | Phase 1/2 Study of CISH Inactivated TILs in the Treatment of NSCLC |
Not yet Recruiting |
Carcinoma, Non-Small-Cell Lung; Metastatic Non Small Cell Lung Cancer; Stage IV Non-small Cell Lung Cancer; Squamous Cell Lung Cancer; Adenocarcinoma of Lung; Large Cell Lung Cancer |
CISH Inactivated TIL | I/II | 70 | Intima Bioscience, Inc. |
| NCT04244656 | A Safety and Efficacy Study Evaluating CTX120 in Subjects With Relapsed or Refractory Multiple Myeloma |
Active, not Recruiting |
Multiple Myeloma | CTX120 | I | 26 | CRISPR Therapeutics AG; CRISPR Therapeutics |
| NCT03655678 | A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion- Dependent β-Thalassemia |
Active, not Recruiting |
Beta-Thalassemia | CTX001 | II/III | 45 | Vertex Pharmaceuticals Incorporated; CRISPR Therapeutics |
| NCT05477563 | Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent #- Thalassemia and Severe Sickle Cell Disease |
Recruiting | Beta-Thalassemia; Thalassemia; Sickle Cell Disease |
CTX001 | III | 12 | Vertex Pharmaceuticals Incorporated; CRISPR Therapeutics |
| NCT04438083 | A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory Renal Cell Carcinoma (COBALT-RCC) |
Recruiting | Renal Cell Carcinoma | CTX130 | I | 107 | CRISPR Therapeutics AG; CRISPR Therapeutics |
| NCT04502446 | A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory T or B Cell Malignancies (COBALTLYM) |
Recruiting | T Cell Lymphoma | CTX130 | I | 45 | CRISPR Therapeutics AG; CRISPR Therapeutics |
| NCT04035434 | A Safety and Efficacy Study Evaluating CTX110 in Subjects With Relapsed or Refractory B-Cell Malignancies (CARBON) |
Recruiting | B-cell Malignancy; Non- Hodgkin Lymphoma; B-cell Lymphoma; Adult B Cell ALL |
CTX110 | I | 143 | CRISPR Therapeutics AG; CRISPR Therapeutics |
| NCT04637763 | CRISPR-Edited Allogeneic Anti-CD19 CAR-T Cell Therapy for Relapsed/Refractory B Cell Non- Hodgkin Lymphoma |
Recruiting | Lymphoma, Non-Hodgkin; Relapsed Non Hodgkin Lymphoma; Refractory B-Cell Non-Hodgkin Lymphoma; |
CB-010 | I | 50 | Caribou Biosciences, Inc. |
| NCT03745287 | A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease |
Active, not Recruiting |
Sickle Cell Disease; Hematological Diseases; Hemoglobinopathies |
CTX001 | II/III | 45 | Vertex Pharmaceuticals Incorporated; CRISPR Therapeutics |
| NCT03728322 | iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations |
Unknown Status |
Thalassemia | iHSCs treatment | early I | 12 | Allife Medical Science and Technology Co., Ltd. |
| NCT04925206 | A Safety and Efficacy Study Evaluating ET-01 in Subjects With Transfusion Dependent β-Thalassaemia |
Active, not Recruiting |
Transfusion Dependent Beta- Thalassaemia |
ET-01 | I | 8 | EdiGene (GuangZhou) Inc. |
| NCT04557436 | TT52CAR19 Therapy for Bcell Acute Lymphoblastic Leukaemia (B-ALL) |
Recruiting | B Acute Lymphoblastic Leukemia |
PBLTT52CAR19 | I | 10 | Great Ormond Street Hospital for Children NHS Foundation Trust; University College, London |
| NCT03747965 | Study of PD-1 Gene-knocked Out Mesothelin-directed CAR-T Cells With the Conditioning of PC in Mesothelin Positive Multiple Solid Tumors |
Unknown Status |
Solid Tumor, Adult | Mesothelin- directed CAR-T Cells |
I | 10 | Chinese PLA General Hospital |
| NCT05565248 | An Open-Label, FIH Study Evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With T1D |
Not yet Recruiting |
Diabetes Mellitus, Type 1; Metabolic Disease; Glucose Metabolism Disorders; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases |
VCTX211 | I/II | 40 | CRISPR Therapeutics AG; ViaCyte; CRISPR Therapeutics |
| NCT03166878 | A Study Evaluating UCART019 in Patients With Relapsed or Refractory CD19+ Leukemia and Lymphoma |
Unknown Status |
B Cell Leukemia; B Cell Lymphoma |
UCART019 | I/II | 80 | Chinese PLA General Hospital |
| NCT05210530 | An Open-Label, FIH Study Evaluating the Safety and Tolerability of VCTX210A Combination Product in Subjects With T1D |
Recruiting | Diabetes Mellitus, Type 1; Metabolic Disease; Glucose Metabolism Disorders; Endocrine System Diseases; Autoimmune Diseases; Immune System Diseases |
VCTX210A unit | I | 10 | CRISPR Therapeutics AG; ViaCyte; CRISPR Therapeutics |
| NCT05356195 | Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants with Transfusion-Dependent #- Thalassemia (TDT) |
Recruiting | Beta-Thalassemia; Thalassemia; Sickle Cell Disease |
CTX001 | III | 12 | Vertex Pharmaceuticals Incorporated; CRISPR Therapeutics |
| NCT05329649 | Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) |
Recruiting | Sickle Cell Disease; Hydroxyurea Failure; Hemoglobinopathies; Hematological Diseases |
CTX001 | III | 12 | Vertex Pharmaceuticals Incorporated; CRISPR |
| NCT03081715 | PD-1 Knockout Engineered T Cells for Advanced Esophageal Cancer |
Completed | Esophageal Cancer | PD-1 Knockout T Cells | N/A | 16 | Hangzhou Cancer Hospital; Anhui Kedgene Biotechnology Co.,Ltd |
| NCT05144386 | Study of EBT-101 in Aviremic HIV-1 Infected Adults on Stable ART |
Recruiting | HIV-1-infection | EBT-101 | I | 9 | Excision BioTherapeutics |
| NCT03398967 | 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 |
Unknown Status |
B Cell Leukemia; B Cell Lymphoma |
Universal Dual Specificity CD19 and CD20 or CD22 CAR-T Cells |
I/II | 80 | Chinese PLA General Hospital |
| NCT04601051 | Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv- PN) and Patients With Transthyretin Amyloidosis- Related Cardiomyopathy (ATTR-CM) |
Recruiting | Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy; Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy; Wild-Type Transthyretin Cardiac Amyloidosis |
NTLA-2001 | I | 74 | Intellia Therapeutics |
| NCT04819841 | Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease |
Recruiting | Sickle Cell Disease | GPH101 | I/II | 15 | Graphite Bio, Inc. |
| NCT05066165 | Study Investigating NTLA- 5001 in Subjects with Acute Myeloid Leukemia |
Active, not Recruiting |
Acute Myeloid Leukemia | NTLA-5001 | I/II | 54 | Intellia Therapeutics |
| NCT04976218 | TGF#R-KO CAR-EGFR T Cells in Previously Treated Advanced EGFR- positive Solid Tumors |
Recruiting | Solid Tumor, Adult; EGFR Overexpression |
Biological: TGFβR-KO CAREGFR T Cells |
I | 30 | Chinese PLA General Hospital |
| NCT02793856 | PD-1 Knockout Engineered T Cells for Metastatic Nonsmall Cell Lung Cancer |
Completed | Metastatic Non-small Cell Lung Cancer |
PD-1 Knockout T Cells | I | 12 | Sichuan University; Chengdu MedGenCell, Co., Ltd. |
| NCT04767308 | Safety and Efficacy of CT125A Cells for Treatment of Relapsed/Refractory CD5+ Hematopoietic Malignancies |
Not yet Recruiting |
CD5+ Relapsed/ Refractory Hematopoietic Malignancies; Chronic Lymphocytic Leukemia (CLL); Mantle Cell Lymphoma (MCL); Diffuse Large B-cell Lymphoma (DLBCL); Follicular Lymphoma (FL); Peripheral T-cell Lymphomas (PTCL) |
CT125A cells | early I | 18 | Huazhong University of Science and Technology; Shanghai IASO Biotechnology Co., Ltd |
| NCT04417764 | TACE Combined With PD-1 Knockout Engineered T Cell in Advanced Hepatocellular Carcinoma. |
Unknown Status |
Advanced Hepatocellular Carcinoma |
PD-1 knockout engineered T Cells |
I | 10 | Central South University |
| NCT05397184 | Study of Base Edited CAR7 T Cells to Treat T Cell Malignancies (TvT CAR7) |
Recruiting | Relapsed/Refractory T-cell Acute Lymphoid Leukaemia |
Cryopreserved BE CAR7 T cells (BE752TBCCLCAR7PBL) |
I | 10 | Great Ormond Street Hospital for Children NHS Foundation Trust; UCL Great Ormond Street Institute of Child Health; Medical Research Council |
| NCT05143307 | Long-Term Follow-Up Study of HIV-1 Infected Adults Who Received EBT-101 |
Enrolling by invitation |
HIV-1-infection | EBT-101 | I | 9 | Excision BioTherapeutics |
| NCT05120830 | NTLA-2002 in Adults With Hereditary Angioedema (HAE) |
Recruiting | Hereditary Angioedema | NTLA-2002 | I/II | 55 | Intellia Therapeutics |
| NCT05514249 | Treatment of a Single Patient With CRD- TMH-001 |
Active, not Recruiting |
Duchenne Muscular Dystrophy |
CRD-TMH-001 | I | 1 | Cure Rare Disease, Inc; University of Massachusetts, Worcester |
| NCT03044743 | PD-1 Knockout EBV-CTLs for Advanced Stage Epstein- Barr Virus (EBV) Associated Malignancies |
Unknown Status |
Stage IV Gastric Carcinoma; Stage IV Nasopharyngeal Carcinoma; T-Cell Lymphoma Stage IV; T-Cell Lymphoma Stage IV; Stage IV Diffuse Large B- Cell Lymphoma |
Fludarabine; Cyclophosphamide; Interleukin-2 |
I/II | 20 | Yang Yang; The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School |
| NCT05444894 | EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT) |
Recruiting | Transfusion Dependent Beta Thalassemia; Hemoglobinopathies; Thalassemia Major; Thalassemia Intermedia |
EDIT-301 | I/II | 6 | Editas Medicine, Inc. |
| NCT03872479 | Single Ascending Dose Study in Participants With LCA10 |
Recruiting | Leber Congenital Amaurosis 10; Inherited Retinal Dystrophies; Eye Diseases, Hereditary; Retinal Disease; Retinal Degeneration; Retinal Degeneration; Eye Disorders Congenital |
EDIT-101 | I/II | 34 | Editas Medicine, Inc. |
N/A, Not Applicable.