BRISBANE, Calif., April 15, 2019 /PRNewswire/ -- Sangamo
Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company,
today announced that Sangamo scientists and collaborators will
present data from the Company's clinical and preclinical pipeline
at the 22nd Annual Meeting of the American Society of
Gene & Cell Therapy (ASGCT) being held April 29th to May 2nd, 2019 in Washington, D.C.
Abstracts accepted for presentation highlight data from the
Company's gene therapy program in Fabry disease; ex vivo
gene-edited cell therapy programs in hemoglobinopathies being
developed in partnership with Sanofi; and preclinical programs for
CNS diseases using Sangamo's gene regulation approach, which
include tauopathies, C9ORF72-linked ALS in collaboration with
Pfizer, and Huntington's disease in collaboration with Takeda.
"Sangamo's strong scientific presence at ASGCT demonstrates the
breadth of our therapeutic pipeline and our expertise in innovative
genomic medicines research and development," said Adrian Woolfson, M.D., Ph.D., Sangamo's
Executive Vice President of Research and Development. "Through our
pioneering work in developing zinc fingers for ex vivo and
in vivo genome editing, we've gained invaluable insights
into AAV manufacturing and delivery, as well as gene editing
precision, efficiency, and specificity. We're now applying these
insights to our integrated portfolio of programs in both gene
therapy and ex vivo cell therapy as well as to our new zinc
finger protein transcription factor gene regulation platform, which
will be showcased at this year's meeting."
ASGCT Annual Meeting presentations
Gene Therapy
- Liver-Targeted AAV Gene Therapy Vectors Produced at Clinical
Scale Result in High, Continuous Therapeutic Levels of α-GalA
Enzyme Activity and Effective Substrate Reduction in a Mouse Model
of Fabry Disease – Abstract #794
Session: Metabolic,
Storage, Endocrine, Liver and Gastrointestinal Diseases II
Poster Presentation – Wednesday, May
1; 5:00PM ET
Ex Vivo Gene-Edited Cell Therapy
- Zinc Finger Nuclease-Mediated Disruption of the BCL11A
Erythroid Enhancer in Human Hematopoietic Stem and Progenitor Cells
Results in Enriched Bialleleic Editing with Highly Replicable and
Precise On-Target Small Indels and Allele-Additive Increases in
Fetal Hemoglobin – Abstract #972
Session: Gene Editing
for Red Blood Cell Disorders
Oral Presentation –
Thursday, May 2; 11:00AM ET
Zinc Finger Nuclease Engineering
- Optimizing Nuclease Specificity via Catalytic Domain
Engineering Enables Complete Gene Modification with No Detectable
Off-Targets – Abstract #133
Session: Measuring and
Mitigating Genotoxicity of Genome Editing
Oral Presentation
– Monday, April 29; 4:00PM ET
Gene Regulation
- Evaluation of Programmable Zinc Finger Protein Transcription
Factors for the Efficient Reduction of Tau in the Nonhuman Primate
Brain – Abstract #62
Session: Gene Silencing
Approaches
Oral Presentation – Monday, April 29; 10:45AM
ET
- Repression of mHTT Expression in Huntington's Disease Mouse
Models by AAV-Mediated Expression of Zinc-Finger Protein-Repressor
Transgene – Abstract #63
Session: Gene Silencing
Approaches
Oral Presentation – Monday, April 29; 11:00AM
ET
- Selective Repression of C90RF72 Repeat Expansion-Containing
Transcripts for the Treatment of ALS – Abstract #555
Session: Neurologic Diseases II
Poster Presentation –
Tuesday, April 30; 5:00PM ET
Invited Presentations at Scientific Symposia
Sangamo's research and development team and clinical
collaborators have additionally been invited to participate in a
variety of special workshops and scientific symposia. Dr.
Ed Rebar, Sangamo's chief technology
officer, will present an overview of preclinical and clinical
programs in the Gene Editing Workshop program and later in the week
will discuss stem cell modification for the treatment of
hemoglobinopathies. Dr. Paul
Harmatz, a professor at UCSF Benioff Children's Hospital
Oakland and a principle investigator for Sangamo's in vivo
genome editing clinical programs in mucopolysaccharidosis type I
(MPS I) and MPS II, will review clinical data presented earlier
this year at the 2019 WORLDSymposium. Dr. Sagar Vaidya, a senior medical director at
Sangamo, will discuss operational aspects of genomic medicine
clinical trials.
- Therapeutic Genome Editing and Gene Regulation Using
Designed Zinc Finger Proteins – Ed
Rebar, Ph.D., Sangamo Therapeutics
Session: Gene
Editing Workshop
Invited Talk – Sunday, April 28; 5:00PM
ET
- Clinical Trials of Genome-editing (ZFN) for MPS I and MPS II
– Paul Harmatz, M.D., UCSF Benioff
Children's Hospital Oakland
Session: Novel Strategies for
Lung and GI Tract-directed Genetic Therapies – Organized by the
Respiratory & GI Tract Gene & Cell Therapy
Committee
Invited Talk – Monday,
April 29; 9:00AM ET
- Gene Modification of Hematopoietic Stem Cells for Research
and Treatment of Hemoglobinopathies – Ed Rebar, Ph.D., Sangamo Therapeutics
Session: Genetic Modification of Stem Cells – Organized by the
Stem Cell Committee
Invited Talk – Wednesday, May 1; 9:30 AM
ET
- IRB and Patient Safety 5 Things You Must Know to Start a
Human Gene Therapy Trial – Sagar
Vaidya, M.D., Ph.D., Sangamo Therapeutics
Session: Getting to the Finish Line: Market Success – Organized
by the Clinical Trials and Regulatory Affairs Committee
Invited Talk – Thursday, May 2;
8:30AM ET
All abstracts for the ASGCT Annual Meeting are available
online at 2019 ASGCT Annual Meeting Abstracts.
About Sangamo Therapeutics
Sangamo
Therapeutics, Inc. is focused on translating ground-breaking
science into genomic medicines with the potential to transform
patients' lives using gene therapy, ex vivo
gene-edited cell therapy, in vivo
genome editing, and gene regulation. For more
information about Sangamo, visit 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 third
parties for clinical trial supply and conduct of clinical trials,
the lengthy and uncertain regulatory approval process,
uncertainties related to the timing of initiation, enrollment and
completion of clinical trials, and whether clinical trial results
will validate and support the safety and efficacy of our product
candidates. 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 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. These risks and uncertainties are described more fully in
Sangamo's Annual Report on Form 10-K and its Current Report on Form
8-K 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.