Sangamo Therapeutics Presents Next-Generation Modular Integrase Technology Engineered to Enable Large-Scale Genome Editing
09 Mayo 2024 - 7:05AM
Business Wire
Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines
company, today presented pre-clinical data showcasing its novel
next-generation integrase technology engineered to enable
large-scale genome editing. Building on Sangamo’s deep expertise in
protein-DNA interactions derived from its zinc finger platform, the
Modular Integrase (MINT) platform is a versatile, protein-guided
genome editing method designed to integrate large sequences of DNA
into the genome to potentially treat – with a single medicine –
patients who have unique mutations in the same gene.
“Precisely integrating large DNA constructs into desirable
chromosomal sites has traditionally been challenging for the field.
We are excited that our new MINT platform potentially addresses
this long-standing problem and would allow us to pursue new
indications by inserting or replacing entire genes,” said Greg
Davis, Ph.D., 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
capitalizes on the strength of Sangamo’s structural protein-DNA
engineering capabilities to deliver potential genomic medicines to
the patients who need them most.”
The MINT platform utilizes Bxb1, a serine recombinase, to
integrate large sequences of DNA into the genome and is intended to
avoid double stranded DNA breaks as well as the need for assistance
from ancillary genome editing or DNA-repair modulating cargo. This
flexible approach is cell-type agnostic, has been engineered to be
simpler to manufacture than most other targeted integration
technologies and is compatible with adeno-associated virus (AAV),
lentiviral and lipid nanoparticle (LNP) delivery. With minimal
dependence on cell DNA repair machinery, we believe the MINT
platform carries a reduced translocation risk and our pre-clinical
data have demonstrated high levels of on-target integration in
certain locations.
Based on these initial findings, we believe the MINT platform
could be deployed internally for neurology-focused indications, and
could provide potential new collaboration opportunities, both for
human disease and in agricultural biotech settings.
Oral and poster presentations from Sebastian Arangundy and
Friedrich Fauser will highlight pre-clinical MINT data at the 27th
American Society of Gene & Cell Therapy (ASGCT) Annual Meeting
being held May 7-11, 2024, in Baltimore, MD, and will also be
available on Sangamo’s website on the dedicated ASGCT 2024
presentations page. Additionally, the publication of a manuscript
in bioRxiv by Jeff Miller and team, titled “Systematic Development
of Reprogrammed Modular Integrases Enables Precise Genomic
Integration of Large DNA Sequences,” further detailing the
discovery and potential of the MINT platform, is available on the
Publications page of Sangamo’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 believes that its zinc finger epigenetic
regulators are ideally suited to potentially address devastating
neurological disorders and that its capsid discovery platform can
expand 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.
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 pre-clinical data and the
potential of these technologies to result in the development of
medicines which 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, as
supplemented by Sangamo’s Quarterly Report on Form 10-Q for the
quarter ended March 31, 2024. 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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Inquiries Louise Wilkie ir@sangamo.com media@sangamo.com
Sangamo Therapeutics (NASDAQ:SGMO)
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