RICHMOND, Calif., April 24, 2017 /PRNewswire/ -- Sangamo
Therapeutics, Inc. (NASDAQ: SGMO), the leader in therapeutic genome
editing, announced that data from the Company's therapeutic and
research programs will be presented at the 20th Annual
Meeting of the American Society of Gene & Cell Therapy (ASGCT)
to be held in Washington, D.C.
from May 10-13, 2017.
Sangamo scientists or collaborators will deliver ten oral and
nine poster presentations during the conference. These
presentations will detail data from therapeutic and research
programs for lysosomal storage disorders and other monogenic
diseases, cancer immunotherapy, and central nervous system
disorders, as well as advancements in genome editing technology and
novel delivery modalities. Sangamo scientists and their
collaborators have also been invited to participate in scientific
symposia and educational sessions focused on clinical and research
applications of genome editing.
"Sangamo once again has a very strong presence at ASGCT, with 19
oral and poster presentations," said Dr. Sandy Macrae, Sangamo's chief executive officer.
"These data highlight the breadth of our clinical and early stage
pipeline across genome editing, gene therapy, gene regulation and
cell therapy. With our focus now on the translation of our
groundbreaking science into new genomic therapies that transform
patients' lives, our research and technology programs will continue
to provide new assets for therapeutic development."
The following presentations are scheduled at the ASGCT Meeting
sessions:
Invited Presentations at Scientific Symposia
- C-Suite Executive Panel: Sandy
Macrae, M.B., Ch.B., Ph.D., Sangamo
Therapeutics
Session: Commercialization
Workshop
Panel Discussion – Tuesday,
May 9; 3:15PM
- Preclinical Studies Evaluating Zinc Finger Nuclease-Driven
Genome Editing – Michael C. Holmes,
Ph.D., Sangamo Therapeutics
Session: Clinical Trials
Training Course
Invited Talk – Tuesday, May 9; 9:50AM
- Educational Session Co-Chair: Thomas
Wechsler, Ph.D., Sangamo Therapeutics
Session:
100. Getting Started in Genome Editing
Panel Discussion –
Wednesday, May 10; 8:00AM
- Scientific Symposium Co-Chair: Michael C. Holmes, Ph.D., Sangamo
Therapeutics
Session: 204. Therapeutic Editing of the
Human Genome and Epigenome
Panel Discussion – Thursday, May 11; 8:00AM
- Scientific Symposium Co-Chair: Kathleen Meyer, M.P.H., Ph.D., D.A.B.T., Sangamo
Therapeutics
Session: 300. Clinical Advancement of Gene
Editing – Moving New Science to the Clinic
Panel Discussion
– Friday, May 12; 8:00AM
Lysosomal Storage Disorders
- Liver-Based Expression of the Human alpha-Galactosidase A
Gene in a Murine Fabry Model Results in Continuous High,
Therapeutic Levels of Enzyme Activity and Effective Substrate
Reduction – Abstract #27
Session: 113. Genome Editing and
Integration Analysis in Metabolic and Endocrine
Disorders
Oral Presentation – Wednesday, May 10; 10:45AM
- ZFN-Mediated In Vivo Genome Editing Results in Phenotypic
Correction in MPS I and MPS II Mouse Models – Abstract
#30
Session: 113. Genome Editing and Integration Analysis
in Metabolic and Endocrine Disorders
Oral Presentation –
Wednesday, May 10; 11:30AM
Central Nervous System Disorders
- Sustained Tau Reduction via Zinc Finger Protein
Transcription Factors as a Potential Next-Generation Therapy for
Alzheimer's Disease and Other Tauopathies – Abstract
#24
Session: 112. Genome Editing: Transcriptional
Regulation and Specificity
Oral Presentation – Wednesday, May 10; 12:00PM
Monogenic and Infectious Diseases
- In Vivo ZFN-Mediated Editing of the Mutant SERPINA1 Gene
Results in Spontaneous Liver Repopulation by the Gene-Edited
Hepatocytes and Greatly Decreased Fibrosis in the PiZ Mouse Model
of alpha-1 Antitrypsin Deficiency Liver Disease – Abstract
#511
Session: 341. In Vivo Gene Editing
Oral
Presentation – Friday, May 12;
4:15PM
- Targeted Genome Editing of Recombination Activating Gene 1
to Potentially Treat Severe Combined Immunodeficiency – Abstract
#654
Session: Gene Targeting and Gene Correction
III
Poster Presentation – Friday, May
12; 5:45PM
- Evolution of HIV-1 Resistance to the Fusion Inhibitor
CD34-CXCR4 and Potential Fitness Costs in Consideration of a Phase
1 Clinical Trial – Abstract #657
Session: Hematologic
& Immunologic Diseases III
Poster Presentation –
Friday, May 12; 5:45PM
Technology and Delivery Developments
- New Zinc Finger Nuclease Architectures for Highly Efficient
Genome Engineering in Primary Cells at Large Scale with No
Detectable Off-Target Effects – Abstract #23
Session:
112. Genome Editing: Transcriptional Regulation and
Specificity
Oral Presentation – Wednesday, May 10;
11:45AM
- In Vivo Genome Editing via Non-Viral Delivery of Zinc Finger
Nucleases Results in Supraphysiological Levels of Therapeutic
Proteins in Adult Mice – Abstract #509
Session: 341. In
Vivo Gene Editing
Oral Presentation – Friday, May 12; 3:45PM
- Non-Viral Delivery of Zinc Finger Nucleases Enable Greater
Than 90% Protein Knockdown of Multiple Therapeutic Gene Targets In
Vivo – Abstract #510
Session: 341. In Vivo Gene
Editing
Oral Presentation – Friday,
May 12; 4:00PM
- Ex Vivo Protein Replacement Using Homology Driven Genome
Editing in Human B Cells by Combining Zinc Finger Nuclease mRNA and
AAV6 Donor Delivery – Abstract #750
Session: 412. Ex Vivo
Gene Editing
Oral Presentation – Saturday, May 13; 11:30AM
- A New, Reversed Zinc-Finger Nuclease Structure for
High-Precision Therapeutic Genome Engineering – Abstract
#170
Session: Gene Targeting and Gene Correction
I
Poster Presentation – Wednesday,
May 10; 5:30PM
- Improved In Vitro Assay to Assess Human Serum Neutralization
of AAV Vectors Yields Cell Line-Dependent Results – Abstract
#396
Session: Immunological Aspects of Gene Therapy and
Vaccines II
Poster Presentation – Thursday, May 11; 5:15PM
- Development of a Qualifiable MiSeq Assay for Precise and
Accurate Quantitation of Small Insertions and Deletions (Indels) in
the Human Genome Induced by Sequence-Specific Zinc Finger Nucleases
– Abstract #644
Session: Gene Targeting and Gene
Correction III
Poster Presentation – Friday, May 12; 5:45PM
Applications of Gene Editing in Stem Cells
- In Vivo Selection of Engineered Human CD34+ HSPCs
Using Targeted Gene Integration – Abstract #512
Session:
341. In Vivo Gene Editing
Oral Presentation – Friday, May 12; 4:30PM
- Correction of SCID-X1 by Targeted Genome Editing of
Hematopoietic Stem/Progenitor Cells (HSPC) in a Humanized Mouse
Model – Abstract #747
Session: 412. Ex Vivo Gene
Editing
Oral Presentation – Saturday,
May 13; 10:45AM
- A Novel Gene Therapy Approach of Fanconi Anemia
Hematopoietic Stem Cells Based on NHEJ-Mediated Gene Editing –
Abstract #165
Session: Gene Targeting and Gene Correction
I
Poster Presentation: Wednesday, May
10; 5:30PM
- Towards Clinical Translation of Hematopoietic Stem Cell Gene
Editing for the Correction of SCID-X1 Mutations – Abstract
#163
Session: Gene Targeting and Gene Correction
I
Poster Presentation: Wednesday, May
10; 5:30PM
- HSPC Expansion Drugs Enhance Gene Editing Efficiency in Long
Term Hematopoietic Stem Cells – Abstract #378
Session:
Gene Targeting and Gene Correction II
Poster presentation:
Thursday, May 11; 5:15PM
- Molecular Evidence of Ex Vivo Genome Editing in a Mouse
Model of Immunodeficiency – Abstract #656
Session:
Hematologic & Immunologic Diseases III
Poster
Presentation – Friday, May 12;
5:45PM
All abstracts for the ASGCT meeting are available online at
2017 ASGCT Annual Meeting Abstracts.
About Sangamo Therapeutics
Sangamo
Therapeutics, Inc. is focused on translating ground-breaking
science into genomic therapies that transform patients' lives using
the company's industry leading platform technologies in genome
editing, gene therapy, gene regulation and cell therapy. The
Company is advancing Phase 1/2 clinical programs in hemophilia A
and hemophilia B, and lysosomal storage disorders MPS I and MPS II.
Sangamo has a strategic collaboration with Bioverativ Inc. for
hemoglobinopathies, including beta thalassemia and sickle cell
disease, and with Shire International GmbH to develop therapeutics
for Huntington's disease. In addition, it has established strategic
partnerships with companies in non-therapeutic applications of its
technology, including Sigma-Aldrich Corporation and Dow
AgroSciences. For more information about Sangamo, visit the
Company's website at www.sangamo.com.
Forward Looking Statements
This press release
contains forward-looking statements based on Sangamo's current
expectations. These forward-looking statements include, without
limitation, references relating to presentation of data from
various therapeutic and research programs and the potential of
these programs to transform the lives of patients. These statements
are not guarantees of future performance and are subject to certain
risks, uncertainties and assumptions that are difficult to predict.
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 ZFP Therapeutics,
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 Reports 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
announcement are made as of this date, and Sangamo undertakes no
duty to update such information except as required under applicable
law.
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SOURCE Sangamo Therapeutics, Inc.