TABLE 4.
Clinical trials based on CRISPR/Cas9 application in medicine.
| Condition or disease | Aim | Study phase | Location | Participant number | Status | NCT number |
|---|---|---|---|---|---|---|
| Viral keratitis | Study of the safety, tolerability, and efficacy of a BD111 CRISPR/Cas9 gene-editing therapy | 1/2 | China | 6 | Active, not recruiting | NCT04560790 |
| Blindness eye | ||||||
| HSV infection | ||||||
| Cornea | ||||||
| HPV-related malignant neoplasm | Study of the safety and efficacy TALEN and CRISPR/Cas9 for HPV-related cervical cancer | 1 | China | 60 | Unknown | NCT03057912 |
| Gastrointestinal cancer | Study of the safety and efficacy of genetically engineered, neoantigen-specific tumor-infiltrating lymphocytes (TIL) | 1/2 | United States | 20 | Recruiting | NCT04426669 |
| Pancreatic cancer | ||||||
| Gallbladder cancer | ||||||
| Colon cancer | ||||||
| Esophageal cancer | ||||||
| Stomach cancer | ||||||
| HIV-1 infection | Study of the safety and feasibility of transplantation with CRISPR/Cas9 CCR5 gene-modified CD34+ HSCs for patients with AIDS | N.A | China | 5 | Unknown | NCT03164135 |
| Thalassemia | Study of the safety and efficacy of transplantation of iHSCs in patients with β-thalassemia | Early 1 | China | 12 | Unknown | NCT03728322 |
| Solid tumors | Study of the safety and feasibility of CRISPR/Cas9 mediated PD-1 and TCR gene-knocked out CAR-T cells in patients with mesothelin + tumors | 1 | China | 10 | Recruiting | NCT03545815 |
| Thalassemia | Study of the safety and efficacy of autologous CRISPR/-Cas9 modified CD34+ HSCs using allogeneic CRISPR/Cas9-engineered T cells | 1/2 | International | 45 | Recruiting | NCT03655678 |
| Hematologic diseases | ||||||
| Hemoglobinopathies | ||||||
| Transfusion dependent beta-thalassemia | Study of the safety and efficacy of autologous CRISPR/Cas9 modified CD34+ HSCs using ET-01 | 1 | China | 8 | Not yet recruiting | NCT04925206 |
| Multiple myeloma | Study of the safety and efficacy of allogeneic CRISPR/Cas9-engineered T cell (CTX120) CTX120 in patients with multiple myeloma | 1 | International | 80 | Recruiting | NCT04244656 |
| Renal cell carcinoma | Study of the safety and efficacy of allogeneic CRISPR/Cas9-engineered T cell (CTX130) in patients with renal cell carcinoma | 1 | International | 107 | Recruiting | NCT04438083 |
| Thalassemia | Study of the long-term safety and efficacy of CTX001 | NA | International | 90 | Enrolling by invitation | NCT04208529 |
| Sickle cell disease | ||||||
| B cell leukemia | Study of the safety of the allogenic gene-edited dual-specificity CD19 and CD20 or CD22 CAR-T cells to treat patients with hematological malignancies | 1/2 | China | 80 | Recruiting | NCT03398967 |
| B cell lymphoma | ||||||
| Solid tumor | Study of the feasibility and safety of CRISPR/Cas9 mediated PD-1 gene knocked-out CAR-T cells in patients with mesothelin + tumors | 1 | China | 10 | Unknown | NCT03747965 |
| B-cell malignancy | Study of the safety and efficacy of allogeneic CRISPR/Cas9-engineered T cell (CTX110) in patients with B-cell malignancies | 1 | International | 143 | Recruiting | NCT04035434 |
| NHL | ||||||
| B-ALL | ||||||
| B-ALL | Study of the safety and efficacy of CD19-specific CAR-T cells with edited endogenous HPK1 in patients with CD19+ leukemia or lymphoma | 1 | China | 40 | Recruiting | NCT04037566 |
| Lymphoma | ||||||
| NHL | Study of the safety, efficacy, pharmacokinetics, and immunogenicity of CRISPR/Cas9- engineered anti-CD19 CAR-T cell in patients with B-NHL | 1 | United States | 50 | Recruiting | NCT04637763 |
| B cell leukemia | Study of the safety and tolerability of universal CD19-specific CAR-T cell in patients with CD19+ leukemia and lymphoma | 1/2 | China | 80 | Recruiting | NCT03166878 |
| B cell lymphoma |