RICHMOND, Calif., Dec. 22 Sangamo BioSciences,Inc. (Nasdaq: SGMO), the leading developer of zinc finger DNA-binding proteins(ZFPs), today announced an agreement to provide Pfizer Inc (NYSE: PFE) with aworldwide, non-exclusive license for the use of certain ZFP Nuclease (ZFNs)reagents to permanently eliminate the Glutamine Synthetase (GS) gene inChinese Hamster Ovary (CHO) cell lines and for the use of these ZFN-modifiedcells for clinical and commercial production of therapeutic proteins. Underthe terms of the agreement Sangamo will receive an upfront payment of $3.0million from Pfizer for a fully paid license.
"Pfizer was an early adopter of Sangamo's ZFN technology for CHO cellengineering," said Edward Lanphier, Sangamo's president and CEO. "Ourcolleagues at Pfizer have made fundamental contributions to establish thebreadth and utility of ZFNs in cell line engineering. We are very pleased toestablish this non-exclusive, commercial protein production license providingPfizer with the right to use ZFNs to eliminate the GS gene in CHO cells, awidely used selection marker for the generation of cell lines used for theproduction of recombinant protein pharmaceuticals and monoclonal antibodies.Based upon our ability to design ZFNs to any gene, we believe that this is oneof many future agreements we may establish, applying our ZFN technology in thecommercial production of protein-based pharmaceuticals."
"We are very pleased to enter into this commercial protein productionlicense agreement with Sangamo. Together we've used ZFNs to generate specificGS knockouts in CHO cells to streamline the creation of mAb production celllines," said David Brunner, Vice President, Bioprocess Research & Development,Pfizer Global Biologics. "We have generated significant research and processdevelopment data following application of the ZFN platform technology. ZFNscan be used to eliminate genes and potentially improve culture performance orthe characteristics of therapeutic proteins being manufactured."
"Prior to the development of ZFN technology, methods for gene disruptionwere limited by their efficiency, time to completion, and the potential forconfounding, off-target effects," said Philip Gregory, D.Phil., Sangamo's VicePresident for Research. "We have demonstrated the power and broadapplicability of our ZFN technology in the engineering of living cells inmultiple publications in high-impact, peer-reviewed journals. Earlier thisyear we published work describing a rapid, single-step approach to targetedgene knockout in mammalian cells using ZFNs (Proc Natl Acad Sci U S A.2008;105):5809-14). We have demonstrated that we can achieve a permanent,heritable elimination of a gene giving a true knockout of that gene in a celland all of its progeny. Our ZFN process is simple, rapid and highly specificand does not require marker genes or the permanent insertion of foreign DNA.Moreover, this is not limited to a single gene in a cell; our ZFNs can be usedto generate a cell line in which multiple genes are selectively andspecifically eliminated. We have been working with scientists at Pfizer toestablish that this process is compatible with suspension growth in serum-freeand animal component-free synthetic media which is an important considerationin human therapeutic protein manufacturing. Our work also confirms that ZFNsare highly-specific; we have not observed any negative impact on cell growth,protein production yield or product characteristics."
Terms of the Agreement
Under this agreement, Sangamo will provide a worldwide, fully paid,perpetual, royalty free, non-exclusive, license for the use of certain ZFNreagents for the elimination of the GS gene in Pfizer's CHO cell lines and touse such ZFN-modified CHO cells for clinical and commercial production oftherapeutic protein products. Sangamo will receive an upfront payment of $3.0million from Pfizer which constitutes full and complete payment for thelicense. The license may not be sublicensed although Pfizer may transfer anyGS ZFN-modified CHO cell line to a contract manufacturer solely for suchcontract manufacturer to manufacture Pfizer's therapeutic proteins for Pfizer.
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 and ALS. Other therapeutic developmentprograms are focused on HIV/AIDS, neuropathic pain, cancer, nerve regenerationand monogenic diseases. Sangamo's core competencies enable the engineering ofa class of DNA-binding proteins known as zinc finger DNA-binding proteins(ZFPs). By engineering ZFPs that recognize a specific DNA sequence Sangamo hascreated ZFP transcription factors (ZFP TF(TM)) that can control geneexpression and, consequently, cell function. Sangamo is also developingsequence-specific ZFP Nucleases (ZFN(TM)) for therapeutic gene modification asa treatment for a variety of monogenic diseases, such as X-linked SCID andhemophilia, and for infectious diseases, such as HIV. Sangamo has establishedstrategic partnerships with companies outside of the human therapeutic spaceincluding Dow AgroSciences, Sigma-Aldrich Corporation and several companiesapplying its ZFP technology to enhance the production of proteinpharmaceuticals. For more information about Sangamo, visit the company's website at http://www.sangamo.com/.
This press release may contain forward-looking statements based onPfizer's and Sangamo's current expectations. These forward-looking statementsinclude, without limitation, the application of the ZFN technology in theengineering of living cells and absence of negative effects on ZFN engineeredcells. Actual results may differ materially from these forward-lookingstatements due to a number of factors, including technological challenges,ability of Sangamo and Pfizer to develop commercially viable products andtechnological developments by our competitors. See the company's SEC filings,and in particular, the risk factors described in the company's Annual Reporton Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamoassumes no obligation to update the forward-looking information contained inthis press release.
SOURCE Sangamo BioSciences, Inc.