Sangamo Therapeutics Presents Next-Generation Modular Integrase Technology Engineered to Enable Large-Scale Genome Editing
“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
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
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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
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