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Table 3 Ongoing CRISPR cell therapy clinical trials.

From: The clinical potential of gene editing as a tool to engineer cell-based therapeutics

DiseaseTrial namePhaseCell type editedTarget patientsStatusSponsorCountriesCT number
CancerPD-1 knockout engineered T cells for advanced esophageal cancerIIAutologous T-cellsPatients with recurrent or metastatic oesophageal cancerStudy completed—February 2018 (first posted: March 16, 2017)Hangzhou Cancer HospitalChinaNCT03081715
PD-1 knockout engineered T cells for metastatic non-small cell lung cancerIAutologous T-cellsPatients with stage IV non-small cell lung cancer with measurable lesionsActive, not recruiting—posted June 8, 2016; updated August 5, 2019Sichuan UniversityChinaNCT02793856
PD-1 knockout EBV-CTLs for advanced stage Epstein–Barr virus (EBV) associated malignanciesI/IIAutologous T-cellsPatients with Epstein–Barr virus+ve stage IV malignancies including: gastric carcinoma, nasopharyngeal carcinoma, T-cell lymphoma, adult hodgkin lymphoma, diffuse large B-cell lymphomaRecruiting—posted February 7, 2017; updated May 2, 2017Yang YangChinaNCT03044743
NY-ESO-1-redirected CRISPR (TCRendo and PD1) edited T Cells (NYCE T Cells)IAutologous T-cellsPatients with relapsed refractory multiple myeloma (MM), melanoma, synovial sarcoma, or myxoid/round cell liposarcoma (MRCL)Active, not recruiting—posted January 16, 2018; updated January 6, 2020University of PennsylvaniaUSANCT03399448
Study of CRISPR–Cas9 mediated PD-1 and TCR gene-knocked out mesothelin-directed CAR-T cells in patients with mesothelin positive multiple solid tumorsIAutologous T-cellsPatients with mesothelin positive tumours that have failed at least one standard care chemotherapy for advanced diseaseRecruiting—posted June 4, 2018; updated December 18, 2019Chinese PLA General HospitalChinaNCT03545815
Study of PD-1 gene-knocked out mesothelin-directed CAR-T cells with the conditioning of PC in mesothelin positive multiple solid tumorsIAutologous T-cellsPatients with mesothelin positive tumours that have failed ≥ 1 standard care chemotherapy for advanced disease, particularly: Pancreatic, cholangiocarcinoma and ovarian cancersRecruiting—posted November 20, 2018; updated November 20, 2018Chinese PLA General HospitalChinaNCT03747965
A feasibility and safety study of universal dual specificity CD19 and CD20 or CD22 CAR-T Cell immunotherapy for relapsed or refractory leukemia and lymphomaI/IIAllogeneic T-cellsPatients with relapsed or refractory CD19+ B-cell leukaemia or lymphomaRecruiting—posted January 16, 2018; updated January 16, 2018Chinese PLA General HospitalChinaNCT03398967
A study evaluating UCART019 in patients with relapsed or refractory CD19+ leukemia and lymphomaI/IIAllogeneic T-cellsPatients with relapsed or refractory CD19+ B-cell leukaemia or lymphomaRecruiting—posted May 25, 2017; updated June 23, 2017Chinese PLA General HospitalChinaNCT03166878
A safety and efficacy study evaluating CTX110 in subjects with relapsed or refractory B-cell malignanciesI/IIAllogeneic T-cellsPatients with relapsed or refractory non-hodgkin’s lymphomaRecruiting—posted July 29, 2019; updated December 10, 2019CRISPR Therapeutics AGUSA, AustraliaNCT04035434
CRISPR (HPK1) edited CD19-specific CAR-T cells (XYF19 CAR-T cells) for CD19+ leukemia or lymphomaIAutologous T-cellsPatients with relapsed or refractory CD19+ B-ALL or other B-cell lymphomasRecruiting—posted July 30, 2019; updated July 30, 2019Xijing HospitalChinaNCT04037566
HIVSafety of transplantation of CRISPR CCR5 modified CD34+ Cells in HIV-infected subjects with hematological malignanciesI/IIAutologous CD34+ HSPCsPatients on cART with undetectable viral load and a haematological neoplasmRecruiting—posted May 23, 2017; updated May 23, 2017Affiliated Hospital to Academy of Military Medical SciencesChinaNCT03164135
Transfusion-dependent β-thalassemiaA safety and efficacy study evaluating CTX001 in subjects with transfusion-dependent β-thalassemiaI/IIAutologous CD34+ HSPCsHomozygous β-thalassemia patients (excluding β0/β0 genotype) or compound heterozygotes including β-thalassemia/haemoglobin E (HbE)Recruiting—posted August 31, 2018; updated December 5, 2019Vertex Pharmaceuticals IncorporatedCanada, Germany, UK, USANCT03655678
Sickle cell diseaseA safety and efficacy study evaluating CTX001 in Subjects with severe sickle cell diseaseI/IIAutologous CD34+ HSPCsSickle cell patients with βS/βS genotype and ≥ 2 vaso-occlusive crisis events yearly for past 2 yearsRecruiting—posted November 19, 2018; updated November 21, 2019Vertex Pharmaceuticals IncorporatedUSA, Germany, Italy, Belgium, CanadaNCT03745287