Sangamo Therapeutics, Inc. a genomic medicines company, 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.”
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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.
SOURCE: Businesswire