Clinical Trial

Disease: Human Immunodeficiency Virus Infection, HIV, (NCT02388594)

Disease info:

Human immunodeficiency virus (HIV) is a virus that attacks the immune system by infecting and killing white blood cells known as CD4+ T-cells. CD4+ T-cells represent a vital part of the immune system and untreated HIV infections render patients more vulnerable to dangerous infections by other pathogens, including bacteria and fungi. If the virus remains untreated it can lead to patients developing the life-threatening disease acquired immunodeficiency syndrome (AIDS). Untreated HIV can leave people vulnerable to life-threatening infections. HIV symptoms often manifest as flu-like symptoms such as fever, chills, rash, night sweats, muscle aches, sore throat, and fatigue. Today, antiviral medications can allow people living with HIV to live healthy lives.


In 2021, 36,136 people received an HIV diagnosis in the United States and dependent areas. An estimated 1.2 million people in the United States had HIV at the end of 2021.
Official title:
A Phase I Study of T-Cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases SB-728mR in HIV-Infected Patients, With or Without the CCR5 Delta-32 Mutation, Pre-treated With Cyclophosphamide

United States, Pennsylvania

University of Pennsylvania, Philadelphia, Pennsylvania, United States, 19104

Study start:
Apr. 1, 2015
14 participants
Gene editing method:
ZFN- Zinc Finger Nucleases
Type of edit:
Gene knock-out
C-C motif chemokine receptor 5 (CCR5)
Delivery method:
mRNA electroporated - Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Completed


This is a triple cohort, open-label pilot study of the safety and antiviral activity of a single infusion of autologous CD4+ T cells genetically modified at the CCR5 gene by Zinc Finger Nucleases SB-728mR (ZFN Modified CD4+ T Cells) using electroporated mRNA with or without the prior administration of two different doses of cyclophosphamide.

Experimental: ZFN Modified CD4+ T Cell
ZFN Modified CD4+ T Cell

Last updated: Dec. 28, 2023
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