Table 3 Ongoing CRISPR cell therapy clinical trials.
From: The clinical potential of gene editing as a tool to engineer cell-based therapeutics
Disease | Trial name | Phase | Cell type edited | Target patients | Status | Sponsor | Countries | CT number |
|---|---|---|---|---|---|---|---|---|
Cancer | PD-1 knockout engineered T cells for advanced esophageal cancer | II | Autologous T-cells | Patients with recurrent or metastatic oesophageal cancer | Study completed—February 2018 (first posted: March 16, 2017) | Hangzhou Cancer Hospital | China | NCT03081715 |
PD-1 knockout engineered T cells for metastatic non-small cell lung cancer | I | Autologous T-cells | Patients with stage IV non-small cell lung cancer with measurable lesions | Active, not recruiting—posted June 8, 2016; updated August 5, 2019 | Sichuan University | China | NCT02793856 | |
PD-1 knockout EBV-CTLs for advanced stage Epstein–Barr virus (EBV) associated malignancies | I/II | Autologous T-cells | Patients with Epstein–Barr virus+ve stage IV malignancies including: gastric carcinoma, nasopharyngeal carcinoma, T-cell lymphoma, adult hodgkin lymphoma, diffuse large B-cell lymphoma | Recruiting—posted February 7, 2017; updated May 2, 2017 | Yang Yang | China | NCT03044743 | |
NY-ESO-1-redirected CRISPR (TCRendo and PD1) edited T Cells (NYCE T Cells) | I | Autologous T-cells | Patients 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, 2020 | University of Pennsylvania | USA | NCT03399448 | |
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 | I | Autologous T-cells | Patients with mesothelin positive tumours that have failed at least one standard care chemotherapy for advanced disease | Recruiting—posted June 4, 2018; updated December 18, 2019 | Chinese PLA General Hospital | China | NCT03545815 | |
Study of PD-1 gene-knocked out mesothelin-directed CAR-T cells with the conditioning of PC in mesothelin positive multiple solid tumors | I | Autologous T-cells | Patients with mesothelin positive tumours that have failed ≥ 1 standard care chemotherapy for advanced disease, particularly: Pancreatic, cholangiocarcinoma and ovarian cancers | Recruiting—posted November 20, 2018; updated November 20, 2018 | Chinese PLA General Hospital | China | NCT03747965 | |
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 | I/II | Allogeneic T-cells | Patients with relapsed or refractory CD19+ B-cell leukaemia or lymphoma | Recruiting—posted January 16, 2018; updated January 16, 2018 | Chinese PLA General Hospital | China | NCT03398967 | |
A study evaluating UCART019 in patients with relapsed or refractory CD19+ leukemia and lymphoma | I/II | Allogeneic T-cells | Patients with relapsed or refractory CD19+ B-cell leukaemia or lymphoma | Recruiting—posted May 25, 2017; updated June 23, 2017 | Chinese PLA General Hospital | China | NCT03166878 | |
A safety and efficacy study evaluating CTX110 in subjects with relapsed or refractory B-cell malignancies | I/II | Allogeneic T-cells | Patients with relapsed or refractory non-hodgkin’s lymphoma | Recruiting—posted July 29, 2019; updated December 10, 2019 | CRISPR Therapeutics AG | USA, Australia | NCT04035434 | |
CRISPR (HPK1) edited CD19-specific CAR-T cells (XYF19 CAR-T cells) for CD19+ leukemia or lymphoma | I | Autologous T-cells | Patients with relapsed or refractory CD19+ B-ALL or other B-cell lymphomas | Recruiting—posted July 30, 2019; updated July 30, 2019 | Xijing Hospital | China | NCT04037566 | |
HIV | Safety of transplantation of CRISPR CCR5 modified CD34+ Cells in HIV-infected subjects with hematological malignancies | I/II | Autologous CD34+ HSPCs | Patients on cART with undetectable viral load and a haematological neoplasm | Recruiting—posted May 23, 2017; updated May 23, 2017 | Affiliated Hospital to Academy of Military Medical Sciences | China | NCT03164135 |
Transfusion-dependent β-thalassemia | A safety and efficacy study evaluating CTX001 in subjects with transfusion-dependent β-thalassemia | I/II | Autologous CD34+ HSPCs | Homozygous β-thalassemia patients (excluding β0/β0 genotype) or compound heterozygotes including β-thalassemia/haemoglobin E (HbE) | Recruiting—posted August 31, 2018; updated December 5, 2019 | Vertex Pharmaceuticals Incorporated | Canada, Germany, UK, USA | NCT03655678 |
Sickle cell disease | A safety and efficacy study evaluating CTX001 in Subjects with severe sickle cell disease | I/II | Autologous CD34+ HSPCs | Sickle cell patients with βS/βS genotype and ≥ 2 vaso-occlusive crisis events yearly for past 2 years | Recruiting—posted November 19, 2018; updated November 21, 2019 | Vertex Pharmaceuticals Incorporated | USA, Germany, Italy, Belgium, Canada | NCT03745287 |