On April 18, 2016 Sangamo BioSciences, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, reported that data from several ZFP Therapeutic programs will be presented at the 19th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) to be held in Washington, D.C. from May 4-7, 2016 (Press release, Sangamo BioSciences, APR 18, 2016, View Source [SID:1234511044]).

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Sangamo BioSciences, Inc. (PRNewsFoto/Sangamo BioSciences, Inc.)
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 (Free ASGCT Whitepaper) 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 (Free ASGCT Whitepaper) 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 (Free ASGCT Whitepaper) 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 (Free ASGCT Whitepaper) meeting are available online at 2016 ASGCT (Free ASGCT Whitepaper) Annual Meeting Abstracts.