Clinical Trial

Disease: Human Immunodeficiency Virus Infection, HIV, (NCT02225665)

Disease info:

HIV stands for human immunodeficiency virus. It harms the immune system by destroying the white blood cells that fight infection. This puts at risk for serious infections and certain cancers.

AIDS stands for acquired immunodeficiency syndrome. It is the final stage of infection with HIV. Not everyone with HIV develops AIDS.

Worldwide, there were about 1.7 million new cases of HIV in 2018. About 37.9 million people were living with HIV around the world in 2018, and 23.3 million of them were receiving medicines to treat HIV, called antiretroviral therapy (ART).
Official title:
A Phase 1/2, Open-Label Study to Assess the Safety and Tolerability of Repeat Doses of Autologous T-Cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases in HIV-Infected Subjects Following Cyclophosphamide Conditioning

Study Director: Winson Tang, MD Sangamo BioSciences, Inc.


United States, California

San Francisco, California, United States, 94115


United States, Connecticut

Norwalk, Connecticut, United States, 06850


United States, Florida

Orlando, Florida, United States, 32803


United States, Texas

Austin, Texas, United States, 78705

Dallas, Texas, United States, 75246


Study start:
Aug. 1, 2014
8 participants
Gene editing method:
ZNF-Zinc Finger Nucleases
Type of edit:
Gene knock-out
C-C motif chemokine receptor 5 (CCR5)
Delivery method:
mRNA transfection - Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Completed


The purpose of this study is to evaluate the safety and tolerability of repeat doses of T-cell immunotherapy (SB-728mR-T) following cyclophosphamide conditioning.

CCR5 is a major co-receptor for HIV entry into T-cells. Disruption of CCR5 by zinc finger nuclease (SB-728mR), blocks HIV entry into the T-cells, therefore, protects the T-cells from HIV infection. Safety (primary outcome) and anti-viral effect (secondary outcome) of zinc finger nuclease-mediated CCR5 disrupted autologous T-cells (SB-728mR-T) will be evaluated in the study.

Last updated: Jun. 13, 2023
Search CRISPR Medicine