Publication in Nature Medicine of First In Vivo Data From Lead Therapeutic Program
RICHMOND, Calif., Nov. 4 /PRNewswire-FirstCall/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO - News) announced today the publication of data that demonstrate the utility of zinc finger DNA binding protein transcription factors (ZFP TFs) as a new class of human therapeutics.
The studies presented in the Nature Medicine article, titled "Induction of angiogenesis in a mouse model using engineered transcription factors," were conducted by Frank Giordano, MD, assistant professor of internal medicine and cardiology at Yale University School of Medicine in collaboration with Sangamo scientists and were sponsored by Edwards Lifesciences Corp. (NYSE: EW - News).
"This work represents a major step towards the development of a new generation of genomics-based drug therapies," said Dr. Giordano. "ZFP TFs can be designed to recognize and bind specifically to DNA sequences in any endogenous gene. The inherent generality and versatility of this approach has implications for a number of novel therapies predicated on the regulation of complex biological processes such as angiogenesis. These studies published in Nature Medicine establish for the first time, the feasibility and potential utility of ZFP Therapeutics."
The authors used engineered ZFP TFs designed to up-regulate the expression of the vascular endothelial growth factor A (VEGF A) gene. Increasing the expression of this gene and the protein that it encodes in a mouse resulted in the appearance of new blood vessels, a process known as angiogenesis. The research also demonstrated that the stimulation of angiogenesis by sequence specific ZFP TFs enabled more rapid wound closure than seen in control animals.
"The data published in the Nature Medicine article provide important evidence of the utility of our lead ZFP TF therapeutic program for treating coronary artery (CAD) and peripheral arterial (PAD) diseases," said Edward Lanphier, Sangamo's president and chief executive officer. "The work also highlights important technical advantages of our ZFP TF technology platform. By activating the cellular copy of the VEGF gene using an engineered ZFP TF, we enable the production of all of the natural protein isoforms of VEGF in the same ratios that are normally produced. These studies clearly show that this results in the generation of less leaky blood vessels, which we believe will be a critical advantage for new therapies aimed at stimulating new blood vessel growth in diseased or damaged heart and skeletal muscles."
The first human therapeutic application of ZFP TFs is likely to be the activation of the VEGF A gene to stimulate new blood vessel growth in the heart to treat CAD or in skeletal muscle to treat PAD. These new vessels would have the potential to bypass blocked arteries, thereby improving blood flow to ischemic tissues. Sangamo BioSciences and Edwards Lifesciences are currently working together to develop ZFP TFs targeting the VEGF gene for these types of therapeutic uses.
Nature Medicine is a monthly publication, the goal of which is to create an understanding between the fields of biomedical science and clinical research. The paper describing this work is available as an Advance Online Publication and can be accessed via the Nature Medicine website at nature.com . The print version of the article will appear in the December 2002 issue of the journal... |
