Sangamo BioSciences study shows benefit of engineered ZFP in treatment of artery disease

By Lisa Kerner

Erie, Pa., Jan. 25 - Sangamo BioSciences, Inc. said it has published preclinical efficacy data demonstrating the potential benefit of zinc finger DNA binding protein transcription factors (ZFP TFs) as a new class of human therapeutics for the treatment of severe late-stage peripheral artery disease.

This stage of peripheral artery disease, known as critical limb ischemia, results in limb loss in a significant number of patients, according to a company news release. Peripheral artery disease affects an estimated 8 million to 10 million people in the United States annually.

The data were the result of a study conducted at Yale University School of Medicine. The study was designed to test the efficacy of a ZFP TF engineered to activate the vascular endothelial growth factor (VEGF) gene in a preclinical model of critical limb ischemia.

Researchers used an engineered ZFP TF designed to activate the expression of the VEGF A gene, an important factor in blood vessel growth (angiogenesis). Using a ZFP TF to increase the expression of VEGF A resulted in statistically significant changes in treated limbs in a number of measures of efficacy. Efficacy end-points included decreased gangrene of the limb and overall cell death in the treated muscle, increased cell growth and increased blood vessel density and blood flow, the company said in the release.

"Our study shows that ZFP TF treatment produces all forms of the VEGF protein in oxygen-starved muscles, statistically significant improvement in blood vessel growth and, most importantly, significantly increased blood flow and promotion of limb salvage as measured over a period of four weeks," said Dr. Frank Giordano, who led the study, in the release.

The study was reported in the current issue of the FASEB Journal

Sangamo scientists developed the VEGF ZFP Therapeutic under an agreement with Edwards Lifesciences, which is testing it in a phase 1 human clinical trial for critical limb ischemia at the Duke University Medical Center.

An additional phase 1 trial of the ZFP Therapeutic for the treatment of the earlier stage of peripheral artery disease, intermittent claudication, is underway at the National Heart Lung and Blood Institute at the National Institutes of Health.

"The strength of the data published in the FASEB article was an important factor in the decision to move this ZFP Therapeutic into a human trial in critical limb ischemia, the more severe stage of [peripheral artery disease]," Edward Lanphier, Sangamo president and chief executive officer, said in the release. "The work also highlights important technical advantages of our ZFP TF technology platform over previous approaches to this problem."

Sangamo BioSciences, based in Richmond, Calif., is focused on the research and development of novel DNA-binding proteins for therapeutic gene regulation and modification.

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