Sangamo BioSciences Announces Presentations On ZFP Therapeutic® Programs And Applications At 2016 Annual Meeting Of The American Society Of Gene & Cell Therapy

RICHMOND, Calif., April 18, 2016 /PRNewswire/ -- Sangamo BioSciences, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, announced that data from several ZFP Therapeutic programs will be presented at the 19th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) to be held in Washington, D.C. from May 4-7, 2016.

Eight oral and seven poster presentations will be given by Sangamo scientists and their academic collaborators. These presentations will detail data from Sangamo's therapeutic and research programs and will focus on lysosomal storage disorders and other monogenic diseases, hemoglobinopathies, HIV/AIDS, cancer immunotherapy and advancements in technology, including improvements in modification efficiency. In addition, Sangamo scientists have been invited to present in two scientific symposia focused on clinical applications of genome editing and gene and cell therapeutics targeting the liver.

"The data to be presented at this year's ASGCT Annual Meeting from our therapeutic and research programs cover a wide range of applications and demonstrate the versatility of our zinc finger nuclease genome editing platform and the expertise of our research and development teams," said Edward Lanphier, Sangamo's president and chief executive officer. "Sangamo continues to lead the clinical development of therapeutic genome editing and ASGCT offers an opportunity for us to present the broad capabilities of our highly leverageable technology platform in multiple therapeutic areas."

The following presentations are scheduled at the ASGCT Meeting sessions:

Invited Presentations at Scientific Symposia

• Genome Editing in Primary Human Cells and Organs: Toward the Goal of Engineering Genetic Cures – Michael C. Holmes, Ph.D., Sangamo BioSciences
  Special Symposium on Concepts and Clinical Applications of Genome Editing
  Invited Talk – Wednesday, May 4, 2016
• ZFN-Mediated Genome Editing in the Liver – Towards Correcting Hemophilias and Lysosomal Storage Diseases – Thomas Wechsler, Ph.D., Sangamo BioSciences
  Scientific Symposium: Targeting the Liver with Gene and Cell Therapeutics
  Invited Talk – Wednesday, May 4, 2016

Lysosomal Storage Disorders

• In Vivo Zinc-Finger Nuclease Mediated Iduronate-2-Sulfatase (IDS) Target Gene Insertion and Correction of Metabolic Disease in a Mouse Model of Mucopolysaccharidosis Type II (MPS II) – Abstract #484
  Session: Targeted Genome Editing: In Vivo Genome Editing
  Oral Presentation – Friday, May 6, 2016
• ZFN-Mediated Liver-Targeting Gene Therapy Corrects Systemic and Neurological Disease of Mucopolysaccharidosis Type I – Abstract #485
  Session: Targeted Genome Editing: In Vivo Genome Editing
  Oral Presentation – Friday, May 6, 2016

HIV/AIDS

• CCR5 Gene Edited Hematopoietic Stem Cells Engraft in Diverse Anatomical Locales and Undergo SHIV-Dependent Positive Selection in Nonhuman Primates – Abstract #38
  Session: Targeted Genome Editing: Gene Editing in Hematopoietic Cells
  Oral Presentation – Wednesday, May 4, 2016
• In Vivo Inhibition of HIV-1 in NSG Mice After Transduction of Primary Human T Cells with CXCR4 Conjugated to an HR2 Peptide – Abstract #427
  Session: Immunological Aspects of Gene Therapy I
  Poster Presentation – Thursday, May 5, 2016
• Pre-Clinical Development and Qualification of ZFN-Mediated Disruption of CCR5 Gene Sequences in Human Hematopoietic Stem and Progenitor Cells – Abstract #734
  Session: Targeted Genome Editing: Methods and Technology
  Oral Presentation – Saturday, May 7, 2016

Hemoglobinopathies

• Targeted Gene Addition in CD34+ Cells from Healthy Donors and Fanconi Anemia Patients – Abstract #558
  Session: Targeted Genome Editing III
  Poster Presentation – Friday, May 6, 2016

Cancer Immunotherapy

• Single Chain TCR Gene Editing in Adoptive Cell Therapy for Multiple Myeloma – Abstract #752
  Session: Cancer-Immunotherapy, Cancer Vaccines III
  Oral Presentation – Saturday, May 7, 2016

Monogenic Diseases

• Towards Clinical Translation of Hematopoietic Stem Cell Gene Editing for the Correction of SCID-X1 Mutations – Abstract #37
  Session: Targeted Genome Editing: Gene Editing in Hematopoietic Cells
  Oral Presentation – Wednesday, May 4, 2016
• Correction of SCID-X1 by Targeted Genome Editing of Hematopoietic Stem/Progenitor Cells (HSPC) in the Mouse Model – Abstract #42
  Session: Targeted Genome Editing: Gene Editing in Hematopoietic Cells
  Oral Presentation – Wednesday, May 4, 2016

Technology Developments and other Applications

• Highly Efficient Homology-Driven Genome Editing in Human T Cells with Combined Zinc-Finger Nuclease mRNA and AAV6 Donor Delivery and Improved Efficiency Under Serum-Free Conditions – Abstract #133
  Session: Targeted Genome Editing I
  Poster Presentation – Wednesday, May 4, 2016
• Valproic Acid Treatment Enhances Hematopoietic Stem and Progenitor Cell Multipotency Ex Vivo for Enhanced Long-Term Engraftment of Gene-Modified Cells – Abstract #432
  Session: Immunological Aspects of Gene Therapy I
  Poster Presentation – Thursday, May 5, 2016
• Highly Efficient, ZFN-Driven Knockout of Surface Expression of the T-Cell Receptor and HLA Class I Proteins in Human T-Cells for Enhancing Allogeneic Adoptive Cell Therapies – Abstract #641
  Session: Cancer-Immunotherapy, Cancer Vaccines III
  Poster Presentation – Friday, May 6, 2016
• Enhanced FVIII AAV Vector Cassette Produces Improved Virus Yields and Supraphysiological FVIII Levels In Vivo – Abstract #684
  Session: Hematologic & Immunologic Diseases II
  Poster Presentation – Friday, May 6, 2016
• Genome Editing of Inducible Cell Lines for Scalable Production of Improved Lentiviral Vectors for Human Gene Therapy – Abstract #286
  Session: Vector and Cell Engineering/Manufacturing
  Oral Presentation – Thursday, May 5, 2016
• Characterization of Chromosomal Alterations Using a Zinc-Finger Nuclease Targeting Both the Beta- and Delta-Globin Gene Loci in Hematopoietic Stem/Progenitor cells – Abstract #118
  Session: Targeted Genome Editing I
  Poster Presentation – Wednesday, May 4, 2016

All abstracts for the ASGCT meeting are available online at 2016 ASGCT Annual Meeting Abstracts.

About Sangamo
Sangamo BioSciences, Inc. is focused on Engineering Genetic Cures^® for monogenic and infectious diseases by deploying its novel DNA-binding protein technology platform in therapeutic genome editing and gene regulation. The Company's proprietary In Vivo Protein Replacement Platform™ (IVPRP) approach is focused on monogenic diseases, including hemophilia and lysosomal storage disorders. Based on its proprietary IVPRP approach, Sangamo is initiating Phase 1/2 clinical trials for hemophilia B, the first in vivo genome editing application cleared by the FDA, and MPS I. In addition, Sangamo has a Phase 2 clinical program to evaluate the safety and efficacy of novel ZFP Therapeutics^® for the treatment of HIV/AIDS (SB-728). The Company has also formed a strategic collaboration with Biogen Inc. for hemoglobinopathies, such as sickle cell disease and beta-thalassemia, and with Shire International GmbH to develop therapeutics for Huntington's disease. It has established strategic partnerships with companies in non-therapeutic applications of its technology, including Dow AgroSciences and Sigma-Aldrich Corporation. For more information about Sangamo, visit the Company's website at www.sangamo.com.

ZFP Therapeutic^® is a registered trademark of Sangamo BioSciences, Inc.

This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, references relating to research and development of novel ZFNs and therapeutic applications of Sangamo's ZFP technology platform, the wide ranging applications of Sangamo's ZFP technology, and the versatility of Sangamo's ZFP technology. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the safety, tolerability and efficacy of ZFNs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo's public filings with the Securities and Exchange Commission, including the risk factors described in its Annual Report on Form 10-K and its most recent Quarterly Report on Form 10-Q. Sangamo assumes no obligation to update the forward-looking information contained in this press release.

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SOURCE Sangamo BioSciences, Inc.

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