As a result of the positive findings, Sangamo expects to initiate a phase I clinical trial to test the HIV ZFP therapy in the second half of 2007.
Sangamo said that its ZFP nuclease (ZFN) technology can be used to make human primary T-cells resistant to HIV infection by permanently modifying the DNA sequence encoding CCR5, an essential co-receptor for the entry of HIV into immune cells.
In this latest stage of the company's program to develop a zinc finger DNA-binding protein therapy for HIV/AIDS, the modified human T-cells were injected into mice. The research showed that the cells engraft, and suggested that, in the presence of HIV, the ZFN-modified cells have a selective advantage and evade HIV infection and destruction.
“This is the first presentation of in vivo data from our program to develop a ZFP therapeutic for the treatment of HIV, and it is significant that these animal data confirm our earlier observations in isolated cells,” said Dale Ando, Sangamo's vice president of therapeutic development and chief medical officer.
Edward Lanphier, Sangamo's president and CEO, added: “By administering ZFNs to patients' T-cells, the goal is to provide HIV-infected individuals with a reservoir of healthy and uninfectable immune cells that would be available to combat opportunistic infections and HIV itself.”
Sangamo's data was presented on June 1, 2007 at the 10th Annual Meeting of the American Society of Gene Therapy, which was held in Seattle, Washington.