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Table 2 Gene editing clinical trials

From: From fiction to science: clinical potentials and regulatory considerations of gene editing

  NCT (status) Country/region Sponsor Disease Target/modification Nuclease Delivery
Ex vivo NCT00842634 (completed) US Sangamo Therapeutics HIV CCR5 modified CD4+ T cells ZFN Adenoviral vector
NCT01044654 (completed) US Sangamo Therapeutics HIV CCR5 modified CD4+ T cells ZFN Adenoviral vector
NCT01252641 (completed) US Sangamo Therapeutics HIV CCR5 modified CD4+ T cells ZFN Adenoviral vector
NCT02388594 (completed) US University of Pennsylvania HIV CCR5 modified CD34+ T cells ZFN mRNA electroporation
NCT02500849 (active, not recruiting) US City of Hope Medical Center HIV CCR5 modified CD34+ HSPCs ZFN mRNA electroporation
NCT03617198 (not yet recruiting) US University of Pennsylvania HIV CCR5 modified, C34-CXCR4, CD4 CAR T cells ZFN Not specified
NCT03666871 (not yet recruiting) Not specified Case Western Reserve University HIV CCR5 modified CD4+ T cells ZFN Not specified
NCT03190278 (recruiting) US Cellectis S.A. Acute myeloid leukemia Allogeneic CAR T cells targeting CD123, TCR disruption TALEN Not specified
NCT03164135 (recruiting) China Affiliated Hospital to Academy of Military Medical Sciences (China) HIV-1 CCR5 modified CD34+ HSPCs (from donor) CRISPR/Cas Not specified
NCT03655678 (recruiting) Canada, Europe Vertex Pharmaceuticals Incorporated Beta Thalassemia Autologous CD34+ HSPCs modified at the enhancer of the BCL11A gene CRISPR/Cas9 Ribonucleoprotein electroporation
NCT03728322 (not yet recruiting) Not specified Allife Medical Science and Technology Co., Ltd Beta thalassemia HBB gene correction in patient specific iHSCs CRISPR/Cas9 Not specified
NCT03745287 (recruiting) US, Europe Vertex Pharmaceuticals Incorporated Sickle cell disease Autologous CD34+ HSPCs modified at the enhancer of the BCL11A gene CRISPR/Cas9 Ribonucleoprotein electroporation
NCT03398967 (recruiting) China Chinese PLA General Hospital B cell leukemia, B cell lymphoma Allogeneic CD19 and CD20/22 CAR T cells CRISPR/Cas9 Not specified
NCT03166878 (recruiting) China Chinese PLA General Hospital B cell leukemia, B cell lymphoma Allogeneic CD19-directed CAR T cells; TCR and B2 M disruption CRISPR/Cas9 Not specified
NCT03399448 (recruiting) US University of Pennsylvania Multiple Myeloma, Melanoma, Synovial Sarcoma, Myxoid/Round Cell Liposarcoma Autologous anti-NY-ESO CAR T cells, disruption of TCR and PD-1 CRISPR/Cas mRNA electroporation
NCT03690011 (not yet recruiting) US Baylor College of Medicine T cell Acute Lymphoblastic Leukemia, T cell Acute Lymphoblastic Lymphoma, T-non-Hodgkin Lymphoma Anti-CD7 CAR T cells, CD7 KO CRISPR/Cas9 Not specified
NCT03545815 (recruiting) China Chinese PLA General Hospital Solid tumor PD-1 and TCR KO anti-mesothelin CAR T cells CRISPR/Cas9 Not specified
NCT03747965 (recruiting) China Chinese PLA General Hospital Solid tumor Mesothelin-directed CAR T cells; PD-1 KO CRISPR Not specified
NCT03081715 (completed) China Hangzhou Cancer Hospital Esophageal cancer PD-1 KO T cells CRISPR/Cas9 Not specified
NCT02793856 (active, not recruiting) China Sichuan University Metastatic non-small cell lung cancer PD-1 KO T cells CRISPR/Cas9 Not specified
NCT03044743 (recruiting) China Yang Yang, Nanjing University Medical School EBV positive advanced stage malignancies PD-1 KO EBV-CTL cells CRISPR/Cas9 Not specified
NCT02863913 (withdrawn—no funding) China Peking University Invasive Bladder Cancer Stage IV PD-1 KO T cells CRISPR/Cas9 Not specified
NCT02867345 (withdrawn—no funding) China Peking University Hormone Refractory Prostate Cancer PD-1 KO T cells CRISPR/Cas9 Not specified
NCT02867332 (withdrawn—no funding) China Peking University Metastatic renal cell carcinoma PD-1 KO T cells CRISPR/Cas9 Not specified
In vivo NCT03041324 (recruiting) US Sangamo Therapeutics Mucopolysaccharidosis II Insertion of corrected copy of Iduronate 2-Sulfatase gene into the Albumin locus ZFN AAV
NCT02702115 (recruiting) US Sangamo Therapeutics Mucopolysaccharidosis I Insertion of corrected copy of α-l-iduronidase gene into the Albumin locus ZFN AAV
NCT02695160 (recruiting) US, Europe Sangamo Therapeutics Hemophilia B Insertion of a corrected copy of the factor 9 gene into the albumin locus ZFN AAV
NCT02800369 (active, not recruiting) China Huazhong University of Science and Technology Human papillomavirus-Related malignant neoplasm E7 ZFN Not specified
NCT03226470 (not yet recruiting) China Huazhong University of Science and Technology Human papillomavirus-related malignant neoplasm E6, E7 TALEN Plasmid in gel
NCT03057912 (unknown) China First Affiliated Hospital, Sun Yat-Sen University Human papillomavirus-Related malignant neoplasm E6, E7 TALEN
CRISPR/Cas9
Plasmid in gel
NCT03872479 (not yet recruiting) US Allergan Leber congenital amaurosis 10 CEP290 CRISPR/Cas9 AAV
  1. ClinicalTrials.gov was searched for clinical trials involving meganucleases, ZFNs, TALENs or CRISPR/Cas. Current status from July 2019