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Table 1 Ongoing and completed clinical trials involving ZFN editing of the cell therapy.

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

DiseaseTrial namePhaseCell type editedDelivery and editing agentStatusSponsorCT number
HIVAutologous T-cells genetically modified at the CCR5 gene by zinc finger nucleases SB-728 for HIV (zinc-finger)IAutologous CD4+ T-cellsAdenoviral vector delivery, SB-728Study completed—January 2013 (first posted—February 12, 2009)University of Pennsylvania and Sangamo TherapeuticsNCT00842634
Study of autologous T-cells genetically modified at the CCR5 gene by zinc finger nucleases in HIV-infected subjectsI/IIAutologous CD4+ T-cellsAdenoviral vector delivery, SB-728Study completed—May 2015 (first posted—December 3, 2010)Sangamo TherapeuticsNCT01252641
Phase 1 dose escalation study of autologous T-cells genetically modified at the CCR5 gene by zinc finger nucleases in HIV-infected patientsIAutologous CD4+ T-cellsAdenoviral vector delivery, SB-728Study completed—December 2014 (first posted—January 8, 2010)Sangamo TherapeuticsNCT01044654
Dose escalation study of cyclophosphamide in HIV-infected subjects on HAART receiving SB-728-TI/IIAutologous CD4+ T-cellsAdenoviral vector delivery, SB-728Study completed—July 2017 (first posted March 2, 2012)Sangamo TherapeuticsNCT01543152
Repeat doses of SB-728mR-T after cyclophosphamide conditioning in HIV-infected subjects on HAARTI/IIAutologous CD4+ T-cellsElectroporated SB-728 mRNAStudy completed—June 2018 (first posted August 24, 2014)Sangamo TherapeuticsNCT02225665
A phase I study of T-cells genetically modified at the CCR5 gene by zinc finger nucleases SB-728mR in HIV-infected patientsIAutologous CD4+ T-cellsElectroporated SB-728 mRNAStudy completed—May 2019 (first posted—March 17 2015)University of PennsylvaniaNCT02388594
Safety study of zinc finger nuclease CCR5-modified hematopoietic stem/progenitor cells in HIV-1 infected patientsICD34+ haematopoietic stem/progenitor cellsElectroporated SB-728 mRNAActive, not recruiting—posted July 17, 2015; updated May 1, 2019City of Hope Medical Center, Sangamo Therapeutic, California Institute for Regenerative Medicine (CIRM)NCT02500849
CCR5-modified CD4+ T cells for HIV infection (TRAILBLAZER)—T-cell reinfusion after interfering with lymphocyte binding location of AIDS virus through zinc-finger-nuclease elimination of CCR5 receptorsI/IIAutologous CD4+ T-cellsAdenoviral vector delivery, SB-728Recruiting—posted September 12, 2018; updated July 24, 2019Case Western Reserve UniversityNCT03666871
A pilot study of T cells genetically modified by zinc finger nucleases SB-728mR and CD4 chimeric antigen receptor in HIV-infected subjectsIAutologous CD4+ T- cellsElectroporated SB-728 mRNARecruiting—posted August 6, 2018; updated August 2, 2019University of PennsylvaniaNCT03617198
Long-term follow-up of HIV subjects exposed to SB-728-T or SB-728mR-TILong-term follow-up of HIV-infected subjects who previously received SB-728-T or SB-728mR-T and completed 3 years of post-infusion follow-upEnrolling by invitation, posted December 17, 2019; updated December 20, 2019Sangamo TherapeuticsNCT04201782
Transfusion-dependent β-thalassemiaA study to assess the safety, tolerability, and efficacy of ST-400 for treatment of transfusion-dependent beta-thalassemia (TDT)I/IIAutologous CD34+ haematopoietic stem/progenitor cellsElectroporated BIVV003/ST-400 mRNARecruiting—posted February 14, 2018; updated September 13, 2019Sangamo Therapeutics and Bioverativ Therapeutics Inc.NCT03432364
Sickle cell diseaseA study to assess the safety, tolerability, and efficacy of BIVV003 for autologous hematopoietic stem cell transplantation in patients with severe sickle cell disease (PRECIZN-1)I/IIAutologous CD34+ haematopoietic stem/progenitor cellsElectroporated BIV003/ST-400 mRNARecruiting—posted August 31, 2018; updated January 13, 2020Bioverativ Therapeutics Inc.NCT03653247