Sangamo BioSciences And Collaborators To Present Data On ZFP Therapeutic Applications At Annual Meeting Of The American Society Of Gene And Cell Therapy

RICHMOND, Calif., April 29, 2015 /PRNewswire/ -- Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced that data from clinical, preclinical and research-stage programs focused on the development of ZFP Therapeutics^® will be presented at the 18th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). Twelve oral and six poster presentations, given by Sangamo scientists and their academic collaborators, will detail data from Sangamo's therapeutic and research programs including HIV/AIDS, hemoglobinopathies, lysosomal storage disorders, and other monogenic diseases, cancer immunotherapy and advancements in technology including delivery. Additionally, members of Sangamo's management team and Company scientists will participate in the C-Suite Executive Panel and two Education Sessions. The meeting will be held in New Orleans, LA, from May 13-16, 2015.

"Our ZFP Therapeutic platform features prominently at this year's ASGCT Annual Meeting, the premier forum for the field of gene and cell therapy, with Sangamo-authored presentations selected for the Presidential Symposium as well as the Clinical Trials Spotlight," said Edward Lanphier, Sangamo's president and CEO. "Data will be presented from our clinical and preclinical studies demonstrating the specificity, versatility and maturity of Sangamo's powerful ZFN genome editing platform across a wide range of therapeutic applications."

The following presentations are scheduled at the ASGCT Meeting sessions:

HIV/AIDS

• Adoptive Transfer of ZFN Mediated CCR5 Modified CD4 T-cells (SB-728-T) in HIV Subjects Leads to Long Term Engraftment of HIV Resistant T Memory Stem Cells and Decrease in Size of Latent Reservoir – Abstract #C-7
  Session: Clinical Trials Spotlight
  Oral Presentation – Thursday, May 14, 2015
• A Dose Escalation Study of Cyclophosphamide (CTX) to Enhance SB-728-T Engraftment – Abstract #25 
  Session: Hematologic and Immunologic Diseases: Clinical Trials and Observations
  Oral Presentation – Wednesday, May 13, 2015
• Long-Term, Multilineage Engraftment of Zinc Finger Nuclease-Edited Hematopoietic Stem Cells in Nonhuman Primates – Abstract #684
  Session: Gene Editing and Gene Regulation III
  Oral Presentation – Saturday, May 16, 2015
• Helper-Dependent Ad5/35 Vectors for ZFN Mediated Gene Editing in Hematopoietic Stem Cells – Abstract #123
  Session: Gene Targeting and Gene Correction I
  Poster Session – Wednesday, May 13, 2015

Hemoglobinopathies

• From GWAS to the Clinic: Genome-Editing the Human Bcl11a Erythroid Enhancer for Fetal Globin Elevation in the Hemoglobinopathies – Abstract #53
  Session: Gene Editing and Gene Regulation I
  Oral Presentation – Wednesday, May 13, 2015
• Preclinical Studies for the First Hematopoietic Stem Cell (Hsc) Gene Editing Trial: Phase 1 Study of Beta-Thalassemia with Autologous Transplantation of Zinc Finger Nuclease-Treated Hsc to Upregulate Fetal Hemoglobin – Abstract #239
  Session: Hematologic and Immunologic Diseases I
  Poster Session – Wednesday, May 13, 2015
• Correction of the Sickle-Cell Disease Mutation in Human Hematopoietic Stem/Progenitor Cells – Abstract #115
  Session: Gene Targeting and Gene Correction I
  Poster Session – Wednesday, May 13, 2015

Lysosomal Storage Disorders

• ZFN-Mediated In Vivo Genome Editing Results in Supraphysiological Levels of Lysosomal Enzymes Deficient in Hunter and Hurler Syndrome and Gaucher Disease – Abstract #479
  Session: Gene Editing and Gene Regulation II
  Oral Presentation – Friday, May 15, 2015

Cancer Immunotherapy

• Clinical Scale Zinc Finger Nuclease (ZFN)-Driven Gene-Editing of PD-1 in Tumor Infiltrating Lymphocytes (TIL) for the Potential Treatment of Metastatic Melanoma – Abstract #77
  Session: Cell Manufacturing, Vector Production, and Biodistribution for Clinical Translation
  Oral Presentation – Wednesday, May 13, 2015
• TCR Gene Editing in a Single Step of T Cell Activation to Redirect T Cell Specificity and Prevent GvHD – Abstract #209
  Session: Cancer – Immunotherapy, Cancer Vaccines I
  Poster Session – Wednesday, May 13, 2015

Monogenic Diseases

• Genome Editing of Primary Human CD34+ Hematopoietic Stem Cells Enables a Safe Harbor Targeted Gene Addition Therapeutic Strategy for Chronic Granulomatous Disease – Abstract #54
  Session: Gene Editing and Gene Regulation I
  Oral Presentation – Wednesday, May 13, 2015
• Targeted Genome Editing in Mouse Hematopoietic Stem/Progenitor Cells (HSPC) to Model Gene Correction of SCID-X1 – Abstract #481
  Session: Gene Editing and Gene Regulation II
  Oral Presentation – Friday, May 15, 2015
• Gene Correction of IL2RG in Human Hematopoietic Stem and Progenitor Cells – Abstract #686
  Session: Gene Editing and Gene Regulation III
  Oral Presentation – Saturday, May 16, 2015
• Targeted Correction and Restored Function of CFTR Gene in Cystic Fibrosis Induced Pluripotent Stem Cells – Abstract #185
  Session: Cardiovascular and Pulmonary Diseases
  Poster Session – Wednesday, May 13, 2015
• Gene Edition for Wiskott-Aldrich Syndrome Gene Therapy – Abstract #342
  Session: Gene Targeting and Gene Correction II
  Poster Session – Thursday, May 14, 2015

Technology Developments and other Applications

• Highly Efficient Targeted Gene Addition in CD34+ Hematopoietic Stem/Progenitor Cells by Combining ZFN mRNA and AAV6 Donor Delivery – Abstract #478
  Session: Presidential Symposium
  Oral Presentation – Friday, May 15, 2015
• AAVHSC Vectors Encoding Zinc-Finger Nucleases Mediate Efficient Targeted Integration at the Human AAVS1 Locus in CD34+ Human Hematopoietic Stem Cells – Abstract #55
  Session: Gene Editing and Gene Regulation I
  Oral Presentation – Wednesday, May 13, 2015
• Targeted Genome Editing of Cell Lines for Improved and Scalable Production of Lentiviral Vectors for Human Gene Therapy – Abstract #6
  Session: RNA Virus Vectors
  Oral Presentation – Wednesday, May 13, 2015

Panel and Discussion Sessions

• Messaging and other strategic tools for successful commercialization in a landscape where zero to few approved therapeutic products exist – Panelist: Edward Lanphier, President and CEO
  Session: C-Suite Executive Panel
  Panel Discussion – Tuesday, May 12, 2015
• Therapeutic Gene Editing – A Biotech Perspective – Speaker: Philip D. Gregory, D.Phil., Senior Vice President, Research and CSO
  Session: Education Session 400: Emerging Field Review: Gene Editing
  Discussion Session – Saturday, May 16, 2015
• Science Funding Challenges: Perspectives from Biopolicy, Bioeconomics and Bioethics – Session Co-Chair: Thomas Wechsler, Ph.D., Scientist III
  Session: Education Session 135: Topical Review
  Discussion Session – Wednesday, May 13, 2015

All abstracts for the ASGCT meeting are available online at 2015 ASGCT Annual Meeting Abstracts.

About Sangamo
Sangamo BioSciences, Inc. is focused on Engineering Genetic Cures^TM for monogenic and infectious diseases by deploying its novel DNA-binding protein technology platform in therapeutic gene regulation and genome editing. The Company has clinical stage programs to evaluate the safety and efficacy of novel ZFP Therapeutics^® for the treatment of HIV/AIDS (SB-728) and beta-thalassemia (SB-BCLmR-HSPC). Sangamo's other therapeutic programs are focused on monogenic and rare diseases. The Company has formed a strategic collaboration with Shire International GmbH to develop therapeutics for hemophilia, Huntington's disease and other monogenic diseases, and with Biogen Inc. for hemoglobinopathies, such as sickle cell disease and beta-thalassemia. It has also 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.

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