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Table 2 TALEN 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
CancerStudy of UCART19 in pediatric patients with relapsed/refractory B acute lymphoblastic leukemia (PALL)IAllogeneic T-cellsPatients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukaemia (B-ALL)Recruiting—posted June 21, 2016; updated October 25, 2019Institut de recherches internationales servierUSA, UK, Belgium, France, SpainNCT02808442
Dose escalation study of UCART19 in adult patients with relapsed/refractory B-cell acute lymphoblastic leukaemia (CALM)IAllogeneic T-cellsPatients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukaemia (B-ALL)Recruiting—posted April 21, 2016; updated October 25, 2019Institut de Recherches Internationales ServierUSA, UK, France, JapanNCT02746952
A study to evaluate the long-term safety of patients with advanced lymphoid malignancies who have been previously administered with UCART19/ALLO-501IAllogeneic T-cellsPatients with advanced lymphoid malignancies dosed with UCART19/ALLO-501 (long-term safety evaluation)Enrolling by invitation—posted April 12, 2016; updated January 7, 2020Institut de Recherches Internationales ServierUSA, Belgium, France, Spain, UKNCT02735083
Safety and efficacy of ALLO-501 anti-CD19 allogeneic CAR T cells in adults with relapsed/refractory large B cell or follicular lymphoma (ALPHA)I/IIAllogeneic T-cellsRelapsed or refractory CD19 positive large B-cell lymphoma or follicular lymphoma patientsRecruiting—posted May 6, 2019; updated January 13, 2020Allogene TherapeuticsUSANCT03939026
Study evaluating safety and efficacy of UCART123 in patients with acute myeloid leukemia (AMELI-01)IAllogeneic T-cellsPatients with CD123 expressing relapsed/refractory AML patients, and in poor-prognosis, newly diagnosed AML patients in the European LeukemiaNet (ELN) adverse genetic risk groupRecruiting—posted June 16, 2017; updated December 6, 2019Cellectis S.A.USANCT03190278
Safety and efficacy of ALLO-715 BCMA allogenic CAR T cells in in adults with relapsed or refractory multiple myeloma (UNIVERSAL) (UNIVERSAL)IAllogeneic T-cellsPatients with relapsed or refractory multiple myeloma (MM) refractory to at least three prior lines of MM therapyRecruting—posted September 18, 2019; updated December 12, 2019Allogene TherapeuticsUSANCT04093596
Phase I study of UCART22 in patients with relapsed or refractory CD22+ B-cell acute lymphoblastic leukemia (BALLI-01)IAllogeneic T-cellsPatients with relapsed or refractory CD22+ B-cell acute lymphoblastic leukaemia (B-ALL)Recruiting—posted November 4, 2019Cellectis S.AUSANCT04150497
Study evaluating safety and efficacy of UCART targeting CS1 in patients with relapsed/refractory multiple myeloma (MELANI-01)IAllogeneic T-cellsPatients with relapsed or refractory multiple myeloma (MM)Recruiting—posted October 29, 2019; updated November 27, 2019Cellectis S.AUSANCT04142619