Sangamo BioSciences has announced that its proprietary zinc finger nuclease (ZFN)-based genome editing technology has been safely engineered to mimic a naturally occurring genetic mutation that provides resistance to HIV infection. SB-728-T is an autologous CD4+ T-cell product in which the gene for CCR5, a co-receptor for HIV entry, is modified via ZFN-mediated genome editing to disrupt the CCR5 protein.

During the Phase 1 open-label study, 12 HIV-infected patients who were well controlled with antiretroviral medications received a single dose of approximately 10 billion ZFN-modified autologous T-cells). Four weeks after the SB-728-T treatment, participants in Cohort 1 (n=6) ceased taking ART for up to 12 weeks and Cohort 2 (n=6) continued with ART throughout the study. The primary objective of the study was to assess safety and tolerability of administration of a single dose of SB-728-T.

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The results showed that modified cells have a favored survival when exposed to HIV, compared to unmodified cells, when ART is discontinued. In the 12-week period, one participant in the ART interruption group reached a decrease in viral load to undetectable levels.

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