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The research phase of the agreement will be conducted in collaborationwith both Sangamo and Sigma-Aldrich, Sangamo's exclusive licensee of ZFNtechnology for high-value research reagents.
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"There is growing appreciation of the value of ZFN technology as a rapid,reliable and highly specific tool for modifying genes in eukaryotic cells andwhole organisms," said David Smoller, Ph.D., President of Sigma-Aldrich'sResearch Biotech Business Unit. "We are excited to be working with Sangamo toprovide Roche with high-value ZFN reagents for the generation of transgenicanimals. ZFN technology promises to enable the generation of a variety oftransgenic models of human disease, expediting drug development andproduction."
"Roche is a leading global healthcare company with an establishedreputation of innovation. We are pleased to provide them with our ZFNtechnology which provides a cutting-edge approach for disease research," saidEdward Lanphier, Sangamo's President and Chief Executive Officer. "Thefrequency and precision of ZFN-mediated genome editing, in combination withthe ability to design ZFNs against potentially any gene, opens up thepossibility of more easily generated transgenic animals of any species."
Zinc finger DNA-binding proteins (ZFPs) are the dominant class ofnaturally occurring transcription factors in organisms from yeast to humans.Transcription factors, which are found in the nucleus of every cell, bind toDNA to regulate gene expression. Though there are many kinds of transcriptionfactors, only ZFPs are amenable to engineering and precise targeting of aparticular gene or genes of interest. ZFNs are engineered forms of ZFPs thatalso contain a nuclease component which can facilitate modification of atarget gene of interest.
"The power of ZFN technology was recently demonstrated in a studypublished in Nature Biotechnology," said Philip Gregory, D.Phil., Sangamo'sVice President for Research. "Using ZFNs in zebrafish, a widely recognizedsystem for human disease modeling and in vivo drug discovery, resulted in thegeneration of fish in which the ZFN-targeted gene has been eliminated or'knocked out'. Apart from the mouse, the majority of other animals includingzebrafish have historically lacked methods for precision targeted mutagenesis.This publication demonstrates that ZFNs present a powerful solution to thisproblem with application across virtually any species for any gene."
Terms of the Agreement
Under this agreement, Sangamo will provide a non-exclusive, worldwideresearch license for the use of its proprietary ZFN technology in thedevelopment of transgenic animals. In this phase Roche will pay researchfees, including research maintenance fees and research event payments. IfRoche elects to exercise its option for an exclusive commercial license itwill owe Sangamo an option license fee, additional payments upon theachievement of certain clinical development events and royalties on sales oftherapeutic and diagnostic products developed by Roche using the ZFN-modifiedanimals.
About the Nature Biotechnology Publication
The work entitled, "Heritable Targeted Gene Disruption in Zebrafish UsingDesigned Zinc Finger Nucleases" was carried out in collaboration with thelaboratory of Sharon Amacher, Ph.D
