Sangamo BioSciences, a clinical research company that develops novel DNA-binding proteins for therapeutic gene regulation and modification, has reported positive preliminary clinical data from the Phase 1 trial (SB-728-902) of its experimental gene therapy to treat HIV.
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The trial aimed at blocking the virus so that patients do not need antiviral drugs, and was designed to evaluate the safety and clinical outcomes of Sangamo’s zinc finger nuclease (ZFN)-generated CCR5-modified, autologous T-cell product (SB-728-T) for the treatment of HIV/AIDS.
Results showed the therapy to be safe when used on six HIV-infected patients on highly active antiretroviral therapy (HAART) with undetectable levels of virus but suboptimal CD4+ T-cell counts.
The treatment functions by modifying a patients’ T-cells to disable a protein called CCR5 (a major co-receptor used by HIV to infect cells of the immune system) that HIV uses to enter the cells.
In the absence of an entry point, HIV is not able to kill off the immune system cells which outlast or eventually overpower the virus.
The trial was led by San Francisco-based Quest Clinical Research director Jacob Lalezari.
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