Sangamo Therapeutics Presents Next-Generation Modular Integrase Technology Engineered to Enable Large-Scale Genome Editing
Sangamo Therapeutics, Inc. presented pre-clinical data on their innovative Modular Integrase (MINT) technology, aimed at large-scale genome editing. The MINT platform utilizes Bxb1 to integrate large DNA sequences into the genome with potential applications in treating patients with unique mutations. The technology is designed to simplify manufacturing, reduce translocation risk, and enable targeted integration without the need for additional genome editing tools. The company views the MINT platform as a valuable tool for neurology-focused indications and potential collaborations in human disease and agricultural biotech.
The MINT platform has the potential to revolutionize genome editing by enabling large-scale DNA integration with reduced translocation risk, high levels of on-target integration, and simplified manufacturing processes.
The technology's versatility and compatibility with various delivery methods, such as AAV, lentiviral, and LNP, make it a promising tool for targeted gene modifications in a cell-type agnostic manner.
The pre-clinical data presented suggest that the MINT platform could open up new opportunities for treating neurological conditions and foster collaborations in the field of human disease and agricultural biotechnology.
Despite the promising pre-clinical data, the actual clinical efficacy and safety of the MINT platform in human trials are yet to be determined, posing a potential risk to the company's future success.
While the technology aims to simplify gene editing processes, any unforeseen challenges in manufacturing or delivery methods could hinder its widespread adoption and commercial viability.
“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
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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Source: Sangamo Therapeutics, Inc.
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