"These data provide good support for the evaluation of our first ZFN-based ZFP Therapeutic in man," commented Dale Ando, M.D., Sangamo's vice president of therapeutic development and chief medical officer. "It was observed more than ten years ago that individuals carrying the natural CCR5-delta32 mutation were highly resistant to infection by HIV. Consequently, a variety of small molecule and antibody approaches have been tested as potential therapeutics. However, a small molecule or antibody approach requires the constant presence of a sufficiently high concentration of drug to block therapeutically relevant numbers of the CCR5 protein, which is present in thousands of copies on the surface of each T-cell and other tissues in the body. We believe that our ZFN technology provides an approach that circumvents the dosing and potential toxicity issues of a systemic therapy. By specifically modifying only CD4 T-cells, the principal target of HIV pathology, in a one-time exposure of the cells to ZFNs, we can generate a population of R5-tropic HIV-resistant T-cells.
We have shown that these ZFN-modified human cells are made permanently resistant to infection by HIV. Furthermore, the cells selectively survive and expand in an animal after HIV infection, providing a reservoir of healthy and uninfectable immune cells. In a patient, such cells could be available to fight both opportunistic infections characteristic of AIDS and HIV itself. The modified cells exhibited the expected properties of normal CD4 T-cells. These data also demonstrate that ZFN-modified human CD4 T-cells can be produced in the quantities required for the translation of this program into the clinic. We intend to file an investigational new drug (IND) application for this ZFP Therapeutic this year and begin a clinical trial as soon as possible."
Data Reported in the ICAAC/IDSA Presentation
The reported results demonstrate that a one-time exposure to
|SOURCE Sangamo BioSciences, Inc|
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