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"Our ZFP Nuclease (ZFN) platform addresses a major problem that has longhampered the development of transgenic models in species other than mice,"said Philip Gregory, Sangamo's vice president of research. "Site-specificmanipulation of the mouse genome via gene targeting revolutionized biology andenabled the generation of mouse cell-lines and transgenic mouse models ofhuman disease. However, until now, and the application of ZFNs, genomes ofother species could not be efficiently modified in a site-specific manner. Thefrequency and precision of ZFN-mediated genome editing, in combination withthe ability to design ZFNs against potentially any gene, opens up thepotential to more easily generate transgenic animals of any species."
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"We are pleased to provide OMT with a non-exclusive, commercial license touse transgenic animals generated using our ZFN technology," said EdwardLanphier, Sangamo's president and chief executive officer. "There is growingappreciation of the value of our technology as a rapid, reliable and highlyspecific tool to modify genes in eukaryotic cells and even whole organisms.As this increases we believe that there will be numerous applications of thetechnology in the generation of a variety of transgenic species as models ofhuman disease and for drug development. "
ZFPs are the dominant class of naturally occurring transcription factorsin organisms from yeast to humans. Transcription factors, which are found inthe nucleus of every cell, bind to DNA to regulate gene expression. Thoughthere are many kinds of transcription factors, only ZFPs are amenable toengineering and precise targeting to a particular gene or genes of interest.ZFNs are engineered forms of ZFPs that also contain a nuclease component,which can induce modification of a target gene of interest.
About Sangamo BioSciences, Inc.
Sangamo BioSciences, Inc. is focused on the research and development ofnovel DNA-binding proteins for therapeutic gene regulation and modification.The most advanced ZFP Therapeutic(TM) development program is currently inPhase 2 clinical trials for evaluation of safety and clinical effect inpatients with diabetic neuropathy. Phase 1 clinical trials are ongoing toevaluate a ZFP Therapeutic for peripheral artery disease. Other therapeuticdevelopment programs are focused on HIV/AIDS, neuropathic pain, cancer, nerveregeneration and monogenic diseases. Sangamo's core competencies enable theengineering of a class of DNA-binding proteins known as zinc finger DNA-binding proteins (ZFPs). By engineering ZFPs that recognize a specific DNAsequence Sangamo has created ZFP transcription factors (ZFP TF(TM)) that cancontrol gene expression and, consequently, cell function. Sangamo is alsodeveloping sequence-specific ZFP Nucleases (ZFN(TM)) for therapeutic genemodification as a treatment for a variety of monogenic diseases, such as X-linked SCID and hemophilia, and for infectious diseases, such as HIV. Sangamohas established several Enabling Technology Agreements with companies to applyits ZFP Technology to enh
