This presentation contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform

Similar documents
Corporate Presentation. March 2018

Q4 and Full Year 2017 Conference Call. February 22, 2018

Immuno-Oncology Program

This presentation contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform

SANGAMO THERAPEUTICS, INC. (Exact name of registrant as specified in its charter)

CNS Gene Regulation Platform

Sangamo Therapeutics Announces Presentations at 2017 Annual meeting of the American Society of Gene & Cell Therapy

Sumiti Jain. [Poster #2003, Tuesday February 13 th ] February 12 th, 2018

Sangamo BioSciences Reports First Quarter 2014 Financial Results

Sangamo BioSciences Reports Third Quarter 2014 Financial Results

This presentation contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform

Sangamo BioSciences Reports Third Quarter 2013 Financial Results

Jefferies 2017 Global Healthcare Conference. Dr Sandy Macrae CEO June 9, 2017

Jefferies 2014 Global Healthcare Conference

We are committed to translating ground-breaking science into genomic therapies that transform patients lives

Jefferies 2016 Healthcare Conference Engineering Genetic Cures

Q Earnings and Corporate Developments. October 31, 2018

Gene Therapy: The Basics. Mark A. Kay MD PhD Dennis Farrey Family Professor Stanford University

Gene Therapy Clinical Pipeline

Total urinary GAGs declined by 51%, dermatan sulfate by 32%, and heparan sulfate by 61% in Cohort 2 at 16 weeks

AdenoVerse Platform for Translational Development of Innovative Gene and Cell Therapies Cell & Gene Therapy World January 18, 2017

2016 Annual Meeting of Stockholders. October 20, 2016

Non-viral Delivery of ZFN mrna Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets

Genome editing: clinical applications FYODOR URNOV

Corporate Presentation OCTOBER 2018

Course Agenda. Day One

SANGAMO THERAPEUTICS, INC. (Exact name of registrant as specified in its charter)

Cell and Gene Therapy Catapult clinical trials database

Sanofi to Acquire Bioverativ A Strategically and Financially Compelling Acquisition in Specialty Care

Russell DeKelver, PhD Sangamo BioSciences, Inc.

About Sarepta Therapeutics

RXi Pharmaceuticals. BioPharm America September 26, 2017 NASDAQ: RXII. Property of RXi Pharmaceuticals

Antibody against Chikungunya virus (mrna-1944)

Welcome to the Webinar! Human Genome Editing: Latest Developments and Advancements

ZIOPHARM Reports Second-Quarter 2016 Financial Results and Provides Update on Recent Activities

Investor Presentation

Corporate Presentation. April 2016

Stem Cells Regenerative Medicine Congress September 15, 2014

Discovery of drugs and drug resistance. Model organisms. Plants and livestock. Cell-based disease modelling. Therapeutics

-- Sarepta strengthens position as a leader in gene therapy; expands rare disease franchise --

First Quarter Trusted by the Life Sciences Industry

(212) Cellectis Media Contact: Philippe Valachs +33(0)

Vertex and Moderna Establish Exclusive Collaboration to Discover and Develop mrna Therapeutics for Cystic Fibrosis

Building a Full-Spectrum Genome Editing Company

Regulatory Pathways for Rare Diseases

Global Leader in Viral Vector Technologies

Corporate Presentation. June 2015

CMC Considerations for Manufacturing of CAR T-Cell Product

Making Hope A Reality bluebird style. November, 2017 Nasdaq : BLUE

Genome Editing Technology - Principle -

Results to be Presented at LDN WORLD Symposium in February Initiation of Repeat-Dose Pompe Study Anticipated in 3Q13

The Cell and Gene Therapy Catapult UK clinical trials database

INTELLIA THERAPEUTICS, INC. (Exact name of registrant as specified in its charter)

The Cell and Gene Therapy Catapult UK clinical trials database

ALLERGAN ENTERS STRATEGIC R&D ALLIANCE WITH EDITAS MEDICINE TO DISCOVER AND DEVELOP CRISPR GENE EDITING PROGRAMS FOR OCULAR DISEASES

Second Quarter 2016 Financial Results. August 4, 2016

Adoptive cellular therapies are based on the administration of live cells into a patient in order for them to serve a therapeutic purpose.

Precigen Company Update

REGENXBIO Inc. Ticker: RGNX

Agios Pharmaceuticals, Inc. (Exact Name of Registrant as Specified in Charter)

Spark Therapeutics, Inc.

Eagle Pharmaceuticals NASDAQ: EGRX

Medical Topics: Gene Therapy. E. Anne Jackson, FSA MAAA July 30, 2018

Topics Covered. FDA s Role in Expediting the Development of Novel Medical Products. How a Regulatory Agency Comes into Existence 3/5/2018

Corporate Overview. June 2017

Welcome to R&D Day! Christine Lindenboom VP, Investor Relations & Corporate Communications

Regulatory Requirements for CRISPR Therapeutics. Bill Lundberg, MD February 2017

Press Release. Interim Data Summary

Stifel Nicolaus Healthcare Conference. September 2012

CHANGES TO A GENE THERAPY MEDICINAL PRODUCT: REGULATORY VIEWS

Together, all nine participants have reduced infusions of factor IX concentrates by 99 percent over cumulative 1,650 days

JUST THE FACTS 1. Nearly 300 Cell and Gene Therapies in Development for A Broad Range of Diseases

Voyager Therapeutics - A Spinout from UMass Gene Therapy and RNAi Technologies

Precigen Company Update

FDA Oversight of Gene Therapy

Pfizer Program in DMD. Beth Belluscio, MD-Ph.D. Pfizer Rare Disease September 9, 2017

Selecta Biosciences Announces Third Quarter 2016 Financial Results and Provides Corporate Update

Global Gene Therapy Market Report -2026

uniqure Completes Strategic Review to Refocus its Pipeline, Reduce Operating Costs and Deliver Long-Term Shareholder Value

PATHWAY TO VALUE CREATION NASDAQ: BNTC ASX: BLT

PATHWAY TO VALUE CREATION NASDAQ: BNTC ASX: BLT. For personal use only

J.P. Morgan Healthcare Conference. Jeffrey Leiden, M.D., Ph.D., Chairman, President and CEO

Thoughts on Gene Editing Francis Collins, M.D. U.S. National Academies Human Gene Editing Committee July 12, 2016

Elena BM Breidenstein, PhD 21 April 2018

Opexa Therapeutics, Inc.

Torreya acted as exclusive financial advisor to Lysogene. About MPS IIIA and LYS-SAF302

-- Expands Sarepta s portfolio to as many as 14 gene therapy programs --

Hemophilia and Gene Therapy

Report of the EMA expert meeting on genome editing technologies used in medicinal product development

Regulatory Perspectives on Gene Therapies for Rare Diseases Rare Diseases Forum Washington, D.C. October 17, 2018

Loxo Oncology Announces Third Quarter 2016 Financial Results

Orchard Therapeutics. Overcoming the complex challenges associated with ex vivo gene therapies. Adrien Lemoine VP Business Development & Operations

Second Quarter 2017 Financial Results. August 8, 2017

Precision Medicine. Presented by:

Personalised Medicine Regulatory Issues

Spark Therapeutics, Inc. (Exact Name of Registrant as Specified in its Charter)

ARQULE AND DAIICHI-SANKYO ENTER INTO STRATEGIC R&D PARTNERSHIP TO PROGRESS NOVEL COMPOUNDS TO TARGET CANCER

Antisense Therapeutics Ltd ASX:ANP January 2017

The Leader in AAV Gene Therapy. A Guide to AAV Gene Therapy for MPS I and II

Transcription:

This presentation contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, as amended. These forward-looking statements include, but are not limited to, the design of clinical trials and expected timing for release of data; the anticipated clinical development milestones and other potential value drivers in the future; the expected benefits of the collaboration with Pfizer and Kite, the expanded capability of Sangamo s technologies; the research and development of novel gene-based therapies and the application of Sangamo s ZFP technology platform to specific human diseases; corporate partnerships; and the potential of Sangamo s genome editing technology to treat genetic diseases. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of various factors and uncertainties. Factors that could cause actual results to differ include, but are not limited to, the dependence on the success of clinical trials of lead programs, the lengthy and uncertain regulatory approval process, uncertainties related to the timing of initiation and completion of clinical trials, whether clinical trial results will validate and support the safety and efficacy of Sangamo s therapeutics, uncertainties related to the initiation and completion of clinical trials, whether clinical trial results will validate and support the safety and efficacy of Sangamo s therapeutics, the reliance on partners and other third-parties to meet their obligations, and the ability to establish strategic partnerships. Further, there can be no assurance that the necessary regulatory approvals will be obtained or that Sangamo and its partners will be able to develop commercially viable gene-based therapeutics. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo s operations and business environments. These risks and uncertainties are described more fully in Sangamo s Annual Report on Form 10-K and Quarterly Reports on Form 10-Q as filed with the Securities and Exchange Commission. Forward-looking statements contained in this presentation are made as of the date hereof, and Sangamo undertakes no obligation to update such information except as required under applicable law. 3

5

6

Discovery Pre-clinical Phase I/II Clinical Phase III / Commercial 7

Therapeutic Area Research Preclinical Phase 1/2 Collaborator Next Milestone MPS I (SB-318) Initial Data: 2018 MPS II (SB-913) Initial Data: late summer 2018 Fabry Disease (ST-920) IND Filing: 2018 Hemophilia A (SB-525) Initial Data: late summer 2018 Hemophilia B (SB-FIX) UK Study Initiation: YE 18 Beta-thalassemia (ST-400) First Subject Enrolled: 1H 18 Sickle Cell Disease (BIVV-003) IND Filing: 2018 Tauopathies ALS/FTLD - C9ORF72 Huntington s Disease Autologous and Allogeneic CAR/TCR/NKR Undisclosed Targets HIV (T cell and Stem Cell)* *only advancing within industry / academic collaborations 8

9

Scope Economics Cell Therapy Oncology T cells and NK cells Ex Vivo genome editing (ZFNs and AAV) CARs, TCRs, and NKRs directed to tumor antigens Autologous and allogeneic products $150M $3.01B Upfront payment In potential R&D and sales-based milestones* Tiered royalties on net sales Solid and liquid tumor types * Aggregated across 10 or more products based on the achievement of certain research, development, regulatory and successful commercialization milestones 10

*FPI First patient in **SCD Sickle Cell Disease 11

Q1 2018 Q1 2017 YE 2017 $in MM, except per share data 12.6 3.4 36.6 R&D 23.5 12.9 65.7 G&A 10.1 7.3 27.2 Operating expenses: $140-150M Total Operating Expenses 33.6 20.2 92.9 Operating Loss (21.0) (16.8) (56.4) Net Loss (20.2) (16.6) (54.6) Net Loss per Share ($0.23) ($0.23) ($0.70) Ending Cash Balance $235M $133M $245M Cash and investments: >$485M 13

14

Therapeutic Area Research Preclinical Phase 1/2 Phase 3 MPS I (SB-318) MPS II (SB-913) Fabry Disease (ST-920) Tauopathies Undisclosed Targets o o o o o o o o 15

*Digital rendering of Sangamo cgmp facility 16

transgene liver cell DNA nucleus therapeutic gene liver cell AAV vectors 18

IND open Study initiated 8 sites active 4 subjects treated US EMA Orphan Drug designation Fast Track designation Orphan Medicinal Product designation 19

zinc finger nucleases transgene + albumin gene AAV vectors Strong albumin promoter H TG H transgene 20

INDs open Studies initiated 5 sites active 4 subjects treated US Orphan Drug designation Fast Track designation Rare Pediatric Disease designation 21

INDs open Studies initiated 6 sites active Multiple patients screening US Orphan Drug designation Fast Track designation Rare Pediatric Disease designation 22

IND open CTA granted Study initiated 4 sites active Plan to initiate U.K. study by YE 2018 US Orphan Drug designation Fast Track designation 23

BCL11A Enhancer Sequence Deletion BCL11A Enhancer Knocked Out 24

IND open Study initiated First subject enrolled expected 1H18 Leverages naturally-occurring, protective mechanism to increase fetal-hemoglobin Highly efficient, precise gene editing; low risk of insertional mutagenesis Non-viral delivery of ZFNs Potentially superior long-term safety profile 25

ZFN ZFN 27

ZFNs 28

Precision Efficiency Specificity Precision Efficiency Specificity Precision Efficiency Specificity 29

Liver-Based Expression of the Human Alpha-Galactosidase A Gene in a Murine Fabry Model Results in Continuous Therapeutic Levels of Enzyme Activity and Effective Substrate Reduction ZFN-Mediated In Vivo Genome Editing Results in Therapeutic Levels of α-galactosidase A and Effective Substrate Reduction in Fabry Knockout Mice Enhancing ZFN Expression Construct and Nuclease Activity Leads to Improvements of In Vivo Genome Editing Platform Global and Tunable Suppression of Zinc Finger Nuclease and ZFP-Transcription Factor Off-Target Activity via Discrete Framework Substitutions Non-Viral Delivery of ZFN mrna Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets Highly Efficient and Specific Multiplexed Gene Editing in T Cells using Enhanced Zinc-Finger Nucleases (ZFNs) Enables Strategic Engineering of Allogeneic T Cell Immunotherapies Designed Zinc Finger Protein Transcription Factors for Single-Gene Regulation Throughout the Central Nervous System 30

32