Strategies for Funding and Developing Stem Cell-Based Therapeutics: A Case Study
Edward Lanphier President & Chief Executive Officer
Sangamo Biosciences
Presented by GTC, a conference production company • 626-256-6405 • www.gtcbio.com
Funding Opportunities in Regenerative Medicine: A Case Study
Edward Lanphier President and Chief Executive Officer
Sangamo BioSciences, Inc.
Stem Cell Summit 2014
April 25, 2014
Sangamo BioSciences
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• Focused on the development of a new class of human therapeutics that function at the DNA level with the goal of “Engineering Genetic Cures”
• Driven by multiple robust technology platforms
– Zinc finger proteins (ZFPs) can be designed to bind to any DNA sequence with singular specificity
– Targeted genome editing and gene regulation
– Broad AAV delivery capabilities, manufacturing and IP
• Broad commercial applications in research reagents, transgenic animals, agriculture, manufacturing and human therapeutics
• Significant partnerships and strong balance sheet
• Dominant intellectual property position
Toolbox for Engineering Genetic CuresTM
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Zinc Finger Protein
ZFP Transcription Factor (ZFP TF)
ZFP Nuclease (ZFN)
• Knockout
• Correct/Add
• Repress
• Activate
Gene Regulation
Domain
Gene Editing Domain
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How does one successfully
fund and develop a platform-
based regenerative medicine
company?
The Challenge……
Sangamo has successfully monetized its ZFP technology platform
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Applications Partners
• Proprietary ZFP Therapeutics® Programs
• Hemoglobinopathies • Sickle Cell Disease • b-thalassemia
• Plant Agriculture • Research Tools • Transgenic Animals • Protein Manufacturing
• Hemophilia A & B • Huntington’s Disease • Two Remaining Targets
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