Table 1

Clinical trials of HIV-infected individuals using ZFN-modified autologous T cells and HSPCs

Clinical trialStatusCohorts/study populationsCell typeZFN delivery methodConditioning regimen
NCT00842634 phase 1 2009-2013 Completed41  On ART, aviremic, CD4 counts >450 CD4 T cells Adenovirus None 
On ART, aviremic, CD4 counts 200-500, ATI 
NCT01044654 phase 1 2009-2014 Completed On ART, aviremic, CD4 counts 200-500 CD4 T cells Adenovirus None 
On ART, aviremic, CD4 counts >500, and CCR5Δ32 heterozygotes, ATI 
NCT01252641 phase 1/2 2010-2015 Completed Not on ART, CD4 counts >500 CD4 T cells Adenovirus None 
NCT01543152 phase 1 2011-2016 Recruiting On ART, aviremic, CD4 counts >500, ATI CD4 T cells, and CD4/CD8 T cells Adenovirus Cytoxan (dose escalation) 
NCT02225665 phase 1/2 2014-2018 Active, not recruiting On ART, aviremic, CD4 counts >500, ATI CD4/CD8 T cells mRNA Cytoxan (1 g/m2
NCT02388594 phase 1 2015-2017 Recruiting On ART, aviremic, CD4 counts >450, ATI CD4 T cells mRNA Cytoxan (1 g/m2
NCT02500849 phase 1 2015-2018 Recruiting On ART, aviremic, CD4 counts 200-500, ATI CD34 HSPC mRNA Busulfan (dose escalation) 
Clinical trialStatusCohorts/study populationsCell typeZFN delivery methodConditioning regimen
NCT00842634 phase 1 2009-2013 Completed41  On ART, aviremic, CD4 counts >450 CD4 T cells Adenovirus None 
On ART, aviremic, CD4 counts 200-500, ATI 
NCT01044654 phase 1 2009-2014 Completed On ART, aviremic, CD4 counts 200-500 CD4 T cells Adenovirus None 
On ART, aviremic, CD4 counts >500, and CCR5Δ32 heterozygotes, ATI 
NCT01252641 phase 1/2 2010-2015 Completed Not on ART, CD4 counts >500 CD4 T cells Adenovirus None 
NCT01543152 phase 1 2011-2016 Recruiting On ART, aviremic, CD4 counts >500, ATI CD4 T cells, and CD4/CD8 T cells Adenovirus Cytoxan (dose escalation) 
NCT02225665 phase 1/2 2014-2018 Active, not recruiting On ART, aviremic, CD4 counts >500, ATI CD4/CD8 T cells mRNA Cytoxan (1 g/m2
NCT02388594 phase 1 2015-2017 Recruiting On ART, aviremic, CD4 counts >450, ATI CD4 T cells mRNA Cytoxan (1 g/m2
NCT02500849 phase 1 2015-2018 Recruiting On ART, aviremic, CD4 counts 200-500, ATI CD34 HSPC mRNA Busulfan (dose escalation) 

Information obtained from website Clinicaltrials.gov and publically reported by sponsors.

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