Engineering Genetic Cures: Genome Editing of Stem Cells

Edward LanphierPresident & Chief Executive Officer

Sangamo Biosciences

Presented by GTC, a conference production company • 626-256-6405 • www.gtcbio.com

“Stem Cell Summit 2016”

Edward LanphierPresident & CEO

Sangamo BioSciences, Inc.

April 26, 2016

Engineering Genetic Cures: Genome Editing of Stem Cells

Agenda

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Topics

Introduction

Genome Editing – Engineering of Zinc Finger Nucleases (ZFNs)

Genome Editing – Therapeutic Strategy / Autologous Cell Therapies

HIV/AIDS – Strategies for a “Functional Cure”

Hemoglobinopathies – Back to the Future… a Partnership Biogen

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 delivery capabilities, manufacturing and IP

• Broad commercial applications in research reagents, transgenic animals, agriculture and human therapeutics

• Significant partnerships and strong balance sheet

• Dominant intellectual property position

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Zinc finger proteins (ZFPs) are a naturally-occurring class of DNA binding protein

α helix (binds DNA)

β sheet

Zinc ion(stabilizes fold)

• Most abundant DNA binding protein in humans

• Alterations of key amino acid positions serve to change the DNA binding specificity

• Enables engineering of individual zinc fingers to bind to specific DNA sequences

• Modular in design

Sangamo Confidential

A Single Finger Provides the Basic DNA Binding Unit

• Short peptide (28 residues)

• Folds via coordination of Zn

• Recognizes 3bp of DNA

• α-helix residues determine triplet binding preference

5’ T A C

3’ A T G

3’

5’

5

Sangamo Confidential

Sangamo’s Platform Uses Two Finger Modules For Design

5’ T A C C C A 3’

3’ A T G G G T 5’

• Enables more specific binding

• Allows one-step assembly of longer ZFPs

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