Queensland's Benitec Heads Toward Its First RNAi  Clinical Trial. (June 24, 2004)

    RNA interference (RNAi) therapy is moving toward clinical trials. While its efficacy has been demonstrated to a limited extent in animals it has not yet been tested in humans.

Now two somewhat different patented approaches look to be nearing initial clinical trials. The Brisbane based Benitec was founded in 1997 to develop and commercialise research from Queensland's Department of Primary Industries (DPI) while Cambridge, Massachusetts based Alnylam Pharmaceuticals came on the scene in 2002. The two firms use different forms of the molecules used to introduce the RNAi but the desired end result is the same, to silence (stop the functioning)  of specifically targeted deleterious genes. According to Nature, Alnylam Pharmaceuticals is "working in diseases such as macular degeneration (progressive vision loss) and also aims to test an RNAi technology in AIDS patients with the condition next year."

Meanwhile Benitec plans to fund a clinical trial for geneticist John Rossi at the City of Hope in Duarte, California who hopes to begin clinical trials next month. HIV infected children will have samples of bone-marrow stem cells removed and treated with specific RNAi. The stem-cells will then be reimplanted in the hope that the treated cells will resist HIV infection.