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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