Table 3. Recent clinical trials using genome editing technologies.
| Trial number (Phase) | Disease | Target gene | Technology | Vector | Start date |
|---|---|---|---|---|---|
| NCT04244656 (I) | Multiple myeloma | BCMA | CRISPR/Cas9 | CAR-T | Jan. 2020 |
| NCT04037566 (I) | Leukemia or Lymphoma | XYF19 | CRISPR/Cas9 | CAR-T | Aug. 2019 |
| NCT04035434 (I/II) | B-Cell malignancies | N/A | CRISPR/Cas9 | CAR-T | Jul. 2019 |
| NCT03728322 (I) | β-thalassemia | HBB | CRISPR/Cas9 | iHSCs | Jan. 2019 |
| NCT03745287 (I/II) | Sickle cell disease | BCL11A | CRISPR/Cas9 | CD34+ hematopoietic stem cells | Nov. 2018 |
| NCT03747965 (I) | Multiple solid tumors | PD-1 | CRISPR/Cas9 | CAR-T | Nov. 2018 |
| NCT03655678 (I/II) | β-thalassemia | BCL11A | CRISPR/Cas9 | CD34+ hematopoietic stem cells | Sep. 2018 |
| NCT03399448 (I) | Multiple myeloma, Melanoma, Synovial sarcoma, Myxoid/Round cell Liposarcoma | PD-1, TCR | CRISPR/Cas9 | T cell | Sep. 2018 |
| NCT03538613 (I/II) | Gastrointestinal epithelial cancer | CISH | CRISPR/Cas9 | CAR-T | May. 2018 |
| NCT03398967 (I/II) | B cell lymphoma | N/A | CRISPR/Cas9 | CAR-T | Jan. 2018 |
| NCT03545815 (I) | Multiple solid tumors | PD-1 and TCR | CRISPR/Cas9 | CAR-T | Jan. 2018 |
| NCT03057912 (I) | HPV-related cervical Intraepithelial neoplasia I | HPV 16 and 18 E7 oncogene | CRISPR/Cas9, TALEN | Plasmid | Jan. 2018 |
| NCT03166878 (I/II) | B cell lymphoma | TCR and B2M | CRISPR/Cas9 | CAR-T | Jun. 2017 |
| NCT03164135 (N/A) | HIV | CCR5 | CRISPR/Cas9 | CD34+ cell | May. 2017 |
| NCT03164135 (N/A) | HIV | CCR5 | CRISPR/Cas9 | Hematopoietic stem cells | May. 2017 |
| NCT03081715 (N/A) | Esophageal cancer | PD-1 | CRISPR/Cas9 | T cell | Mar. 2017 |
| NCT03044743 (I/II) | Epstein-Barr virus associated malignancies | PD-1 | CRISPR/Cas9 | T cell | Apr. 2017 |
| NCT02867345 (I) | Prostate cancer | PD-1 | CRISPR/Cas9 | T cell | Nov. 2016 |
| NCT02867332 (I) | Renal cell carcinoma | PD-1 | CRISPR/Cas9 | T cell | Nov. 2016 |
| NCT02863913 (I) | Bladder cancer | PD-1 | CRISPR/Cas9 | T cell | Sep. 2016 |
| NCT02793856 (I) | Non-small cell lung cancer | PD-1 | CRISPR/Cas9 | T cell | Aug. 2016 |
| NCT04150497 (I) | B-cell acute lymphoblastic leukemia | N/A | TALEN | CAR-T | Oct. 2019 |
| NCT03226470 (I) | Cervical precancerous lesions | HPV16 E6 and E7 DNA | TALEN | T27 and T512 | Jan. 2018 |
| NCT03190278 (I) | Acute myeloid leukemia | N/A | TALEN | CAR-T | Jun. 2017 |
| NCT03653247 (I/II) | Sickle cell disease | BCL11A | ZFN | Hematopoietic stem cell | Jun. 2019 |
| NCT00842634 (I) | HIV | CCR5 | ZFN | T cell | Jan. 2019 |
| NCT03432364 (I/II) | β-thalassemia | BCL11A | ZFN | Hematopoietic stem cells | Mar. 2018 |
| NCT03041324 (I/II) | MPS II | ALB | ZFN | AAV | May. 2017 |
| NCT02702115 (I/II) | MPS I | ALB | ZFN | AAV | May. 2017 |
| NCT02800369 (I) | Cervical precancerous lesions | HPV16 and 18 E7 oncogene | ZFN | N/A | Dec. 2016 |
| NCT02695160 (I) | Hemophilia B | ALB | ZFN | AAV | Nov. 2016 |
| NCT02500849 (I) | HIV | CCR5 | ZFN | Hematopoietic stem cells | Jul. 2015 |
| NCT01252641 (I/II) | HIV | CCR5 | ZFN | T cell | Nov. 2010 |
AAV: adeno-associated virus, CAR: chimeric antigen receptor, CRISPR/Cas9: clustered regularly interspaced short palindromic repeats/CRISPR-associated 9 proteins, HIV: human immunodeficiency virus, HPV: human papillovirus, MPS: mucopolysaccharidosis, N/A: not available, PD-1: programmed cell death-1, TALEN: transcription activator-like effector nucleases, ZFN: zinc finger nucleases