Sangamo Technology Shows Promise in Treating HIV

Sangamo BioSciences Inc., which develops genetic-based therapies, said Monday data shows the company's technology can make certain cells in the immune system resistant to the HIV virus.

The technology, known as zinc finger DNA-binding proteins, acts on the CCR5 gene (a co-receptor that enables HIV to enter and infect cells of the immune system), essentially "knocking-out" the gene.

The results were published online Sunday in the journal Nature Biotechnology.

Sangamo said that while drugs exist that block the binding of HIV to the CCR5 gene, they require a constant high concentration of the drug in the system and have side effects, while its technology requires only a one-time exposure.

Sangamo's ZFNs are designed to permanently modify the DNA sequence covering CCR5. Individuals carrying a naturally occurring mutation of their CCR5 gene have been shown to be resistant to HIV infection.

"The modified cells preferentially survive and expand in an animal after HIV infection, providing a reservoir of healthy and uninfectable immune cells," said Dale Ando, M.D., Sangamo's chief medical officer, in a statement.

Sangamo expects to initiate a clinical trial to evaluate this approach by the end of the year.