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

HIV

Autologous T-cells genetically modified at the CCR5 gene by zinc finger nucleases SB-728 for HIV (zinc-finger)

I

Autologous CD4⁺ T-cells

Adenoviral vector delivery, SB-728

Study completed—January 2013 (first posted—February 12, 2009)

University of Pennsylvania and Sangamo Therapeutics

NCT00842634

Study of autologous T-cells genetically modified at the CCR5 gene by zinc finger nucleases in HIV-infected subjects

I/II

Autologous CD4⁺ T-cells

Adenoviral vector delivery, SB-728

Study completed—May 2015 (first posted—December 3, 2010)

Sangamo Therapeutics

NCT01252641

Phase 1 dose escalation study of autologous T-cells genetically modified at the CCR5 gene by zinc finger nucleases in HIV-infected patients

I

Autologous CD4⁺ T-cells

Adenoviral vector delivery, SB-728

Study completed—December 2014 (first posted—January 8, 2010)

Sangamo Therapeutics

NCT01044654

Dose escalation study of cyclophosphamide in HIV-infected subjects on HAART receiving SB-728-T

I/II

Autologous CD4⁺ T-cells

Adenoviral vector delivery, SB-728

Study completed—July 2017 (first posted March 2, 2012)

Sangamo Therapeutics

NCT01543152

Repeat doses of SB-728mR-T after cyclophosphamide conditioning in HIV-infected subjects on HAART

I/II

Autologous CD4⁺ T-cells

Electroporated SB-728 mRNA

Study completed—June 2018 (first posted August 24, 2014)

Sangamo Therapeutics

NCT02225665

A phase I study of T-cells genetically modified at the CCR5 gene by zinc finger nucleases SB-728mR in HIV-infected patients

I

Autologous CD4⁺ T-cells

Electroporated SB-728 mRNA

Study completed—May 2019 (first posted—March 17 2015)

University of Pennsylvania

NCT02388594

Safety study of zinc finger nuclease CCR5-modified hematopoietic stem/progenitor cells in HIV-1 infected patients

I

CD34⁺ haematopoietic stem/progenitor cells

Electroporated SB-728 mRNA

Active, not recruiting—posted July 17, 2015; updated May 1, 2019

City 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 receptors

I/II

Autologous CD4⁺ T-cells

Adenoviral vector delivery, SB-728

Recruiting—posted September 12, 2018; updated July 24, 2019

Case Western Reserve University

NCT03666871

A pilot study of T cells genetically modified by zinc finger nucleases SB-728mR and CD4 chimeric antigen receptor in HIV-infected subjects

I

Autologous CD4⁺ T- cells

Electroporated SB-728 mRNA

Recruiting—posted August 6, 2018; updated August 2, 2019

University of Pennsylvania

NCT03617198

Long-term follow-up of HIV subjects exposed to SB-728-T or SB-728mR-T

I

Long-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-up

Enrolling by invitation, posted December 17, 2019; updated December 20, 2019

Sangamo Therapeutics

NCT04201782

Transfusion-dependent β-thalassemia

A study to assess the safety, tolerability, and efficacy of ST-400 for treatment of transfusion-dependent beta-thalassemia (TDT)

I/II

Autologous CD34⁺ haematopoietic stem/progenitor cells

Electroporated BIVV003/ST-400 mRNA

Recruiting—posted February 14, 2018; updated September 13, 2019

Sangamo Therapeutics and Bioverativ Therapeutics Inc.

NCT03432364

Sickle cell disease

A 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/II

Autologous CD34⁺ haematopoietic stem/progenitor cells

Electroporated BIV003/ST-400 mRNA

Recruiting—posted August 31, 2018; updated January 13, 2020

Bioverativ Therapeutics Inc.

NCT03653247