Sangamo Therapeutics to Present Neurology-Focused Pre-Clinical Data From Its Epigenetic Regulation, Capsid Delivery and Genome Engineering Platforms at the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)
23 April 2024 - 6:35AM
Business Wire
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine
company, today announced that the American Society of Gene &
Cell Therapy (ASGCT) has accepted 20 Sangamo abstracts for
presentation at the 27th ASGCT Annual Meeting being held May 7-11,
2024, in-person in Baltimore, MD and in a virtual format.
Presentations will focus on the progression of Sangamo’s
neurology-focused pre-clinical pipeline, including data supporting
innovations in zinc finger epigenetic regulation, advances in AAV
capsid engineering, and discovery of next-generation integrase
technology.
“We are thrilled to be showcasing at ASGCT the strong depth of
our neurology pipeline, including various applications for our zinc
finger epigenetic regulation platform, exciting advances in our
capsid delivery technology, and new, potentially transformative
Modular Integrases (MInts), that together reflect our continued
commitment to developing potential neurology genomic medicines for
patients in need,” said Amy Pooler, Head of Research at Sangamo.
“Potent epigenetic regulation in diseases such as prion disease and
tauopathies are complemented by our exceptional AAV delivery
capabilities, a combination that is essential to the development of
effective potential genomic medicine treatments to treat
devastating neurological conditions.”
“These data showcase the deep expertise of our scientific team
in engineering DNA-protein interactions and the broad potential
reach of our innovative pre-clinical approaches, including exciting
new discoveries for site-directed integration of large DNA
sequences via engineered MInts,” said Greg Davis, Head of
Technology at Sangamo. “Using compact, protein-guided integrases
built from Bxb1 variants as a means of targeted integration expands
our genome editing abilities and underscores our drive to develop
technologies that enable potentially transformative new
medicines.”
Data presentations at the ASGCT Annual Meeting include three
oral presentations, two of which detail Sangamo’s potent delivery
capabilities developed through its AAV capsid engineering platform
SIFTER, and one that will discuss a new breakthrough in programming
Bxb1, a large serine recombinase, for the development of MInts to
potentially enable large-scale genome engineering. These data will
showcase a novel AAV capsid, STAC-BBB, that exhibited robust and
widespread central nervous system transduction in cynomolgus
macaques after intravenous delivery, as well as another novel AAV
capsid, STAC-150, engineered to accelerate the discovery of potent
and highly specific epigenetic regulators. Sangamo will also
showcase data highlighting the use of MInts, derived from compact
Bxb1 variants, to integrate large sequences of DNA into the genome
with minimal double-stranded DNA breaks and without the need for
assistance from ancillary genome editing or DNA-repair modulating
cargo.
Additional poster presentations at the ASGCT Annual Meeting will
showcase the use of zinc finger activators and repressors for the
potential treatment of neurological diseases such as prion disease,
tauopathies, Charcot-Marie-Tooth disease type 1A (CMT1A), Dravet
Syndrome, SOD1-mediated amyotrophic lateral sclerosis (ALS),
Phelan-McDermid syndrome, Parkinson’s disease, Angelman syndrome,
and other CNS disorders. Sangamo will also present additional data
from its AAV capsid engineering platform SIFTER and its efforts for
integrase evolution and utilization.
ASGCT Annual Meeting Presentations and
Invited Sessions
Epigenetic Regulation for the Central
Nervous System
- Restoration of Normal Gene and Protein Expression in Mouse and
Human Disease Models of SCN2A Haploinsufficiency Using Zinc Finger
Activators
- Abstract No. 636
- Poster Presentation – May 8; 5:30-7:00 pm ET
- A Zinc Finger Activator Platform to Restore Normal Gene &
Protein Expression in Cellular Models of Dravet Syndrome
- Abstract No. 642
- Poster Presentation – May 8; 5:30-7:00 pm ET
- SNCA Gene Repression Mediated by Zinc Finger Repressors (ZFRs)
as a Therapeutic Approach for Parkinson's Disease
- Abstract No. 1120
- Poster Presentation – May 9; 5:30-7:00 pm ET
- UBE3A Gene Activation Mediated by Zinc Finger Activators (ZFAs)
as a Therapeutic Approach for Angelman Syndrome
- Abstract No. 1121
- Poster Presentation – May 9; 5:30-7:00 pm ET
- Whole CNS Human Tau Knockdown for the Potential Treatment of
Alzheimer’s Disease and Other Tauopathies
- Abstract No. 1126
- Poster Presentation – May 9; 5:30-7:00 pm ET
- SOD1 Gene Repression Mediated by Zinc Finger Repressors (ZFRs)
as a Therapeutic Approach for SOD1-Mediated ALS
- Abstract No. 1597
- Poster Presentation – May 10; 5:30-7:00 pm ET
- PMP22 Gene Repression Mediated by Zinc Finger Repressors (ZFRs)
as a Therapeutic Approach for CMT1A
- Abstract No. 1600
- Poster Presentation – May 10; 5:30-7:00 pm ET
- Shank3 Gene Activation Mediated by Zinc Finger Transcriptional
Activators (ZFA) as a Therapeutic Approach for Phelan-McDermid
Syndrome
- Abstract No. 1605
- Poster Presentation – May 10; 5:30-7:00 pm ET
- Development of a Robust Zinc Finger Activation Platform for
Treatment of Neurological Disorders
- Abstract No. 1609
- Poster Presentation – May 10; 5:30-7:00 pm ET
- Epigenetic Regulation of Human Prion Expression as a Potential
One-Time Treatment for Prion Disease
- Abstract No. 1616
- Poster Presentation – May 10; 5:30-7:00 pm ET
Viral Engineering for the Central
Nervous System
- Identification and Characterization of STAC-BBB, an Engineered
AAV Capsid That Exhibits Widespread Transduction of the Central
Nervous System in Cynomolgus Macaques
- Abstract No. 117
- Oral Presentation – May 8; 4:00-4:15 pm ET
- A Highly Potent Engineered AAV Capsid, STAC-150, Enables
High-Throughput AAV Production and Arrayed Epigenetic Regulator
Screening Directly in Cultured Neurons
- Abstract No. 351
- Oral Presentation – May 10; 5:15-5:30 pm ET
- Optimal Drug Product Presentation and Container Closure
Selection for AAV-Based Genomic Medicines
- Abstract No. 547
- Poster Presentation – May 8; 5:30-7:00 pm ET
- Development of Blood-Brain Barrier Penetrant AAVs through
Receptor-Targeted Capsid Engineering
- Abstract No. 985
- Poster Presentation – May 9; 5:30-7:00 pm ET
- Unraveling Impact of Manufacturing Process-Related Stresses on
AAV Stability, Aggregation, and DNA Release
- Abstract No. 1032
- Poster Presentation – May 9; 5:30-7:00 pm ET
- Process and Formulation Development for a Novel Blood-Brain
Barrier Penetrant AAV Capsid
- Abstract No. 1052
- Poster Presentation – May 9; 5:30-7:00 pm ET
Next-Generation Genome
Engineering
- Directed Evolution of Bxb1 for the Development of Modular
Integrases (MInts)
- Abstract No. 192
- Oral Presentation – May 9; 4:00-4:15 pm ET
- Zinc Finger Mediated Repression and Replacement of MFN2 Leads
to the Rescue of Cellular Disease Phenotype in CMT2A
Patient-Derived Cells
- Abstract No. 637
- Poster Presentation – May 8; 5:30-7:00 pm ET
- Highly Specific Zinc Finger Proteins with Synthetic Target
Sites Enable Self-Regulated Expression of Dosage-Sensitive
Transgenes
- Abstract No. 722
- Poster Presentation – May 8; 5:30-7:00 pm ET
- Site-directed integration of large DNA sequences into
endogenous sites in the human genome using engineered Modular
Integrases (MInts)
- Abstract No. 1680
- Poster Presentation – May 10; 5:30-7:00 pm ET
All abstracts for the ASGCT Annual Meeting are available on
ASGCT’s website.
About Sangamo Therapeutics
Sangamo Therapeutics is a genomic medicine company dedicated to
translating ground-breaking science into medicines that transform
the lives of patients and families afflicted with serious
neurological diseases who do not have adequate or any treatment
options. Sangamo’s zinc finger epigenetic regulators are ideally
suited to potentially address devastating neurological disorders
and Sangamo’s capsid discovery platform is expanding delivery
beyond currently available intrathecal delivery capsids, including
in the central nervous system. Sangamo’s pipeline also includes
multiple partnered programs and programs with opportunities for
partnership and investment. To learn more, visit www.sangamo.com
and connect with us on LinkedIn and Twitter/X.
Sangamo Forward Looking Statements
This press release contains forward-looking statements based on
Sangamo's current expectations. These forward-looking statements
include, without limitation, statements relating to Sangamo’s
technologies, the presentation of data from various therapeutic and
research programs and the potential of these programs to
demonstrate therapeutic benefit and transform the lives of
patients. These statements are not guarantees of future performance
and are subject to certain risks and uncertainties that are
difficult to predict. Factors that could cause actual results to
differ include, but are not limited to, the research and
development process, including the results of clinical trials; the
regulatory approval process for product candidates; and the
potential for technological developments that obviate technologies
used by Sangamo. 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 our Securities and
Exchange Commission filings and reports, including in our Annual
Report on Form 10-K for the year ended December 31, 2023.
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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Investor Relations & Media Inquiries Louise Wilkie
ir@sangamo.com media@sangamo.com
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