Sangamo's ZFN shows efficacy in preclinical HIV study

Sangamo Biosciences, a gene-based therapeutics development firm headquartered in California, USA, say that data from a recent study show that mammalian cells can be made resistant to HIV infection by treatment with the company's proprietary zinc finger DNA-binding nucleases. The results, which were made public at the 45th annual Interscience Conference on Antimicrobial Agents and Chemotherapy, in Washington DC, detailed that the drug was able to cause disruption of the CCR5 gene, which encodes a receptor required by HIV to enter the cell.

Sangamo's president, Edward Lanphier, commented: "individuals with a natural mutation of their CCR5 gene have been shown to be resistant to HIV infection." Mr Lanphier added that several major pharmaceutical firms had attempted to develop compounds which block HIV binding to CCR5, but trials had been halted due to reports of liver toxicity. He concluded that, "using ZFNs to permanently modify the CCR5 gene, specifically in T cells...may have several advantages over the systemic effects of other drugs in development." The company says that, in collaboration with a team led by Carl June at the University of Pennsylvania, it plans to initiate a Phase I clinical trial of the drug in 2006.