Gene-Editing Endonuclease with Nickase Functionality

Many gene-editing strategies involve the use of nucleases to generate targeted double-strand breaks in genomic DNA, which can cause cytotoxicity and off-target effects, limiting clinical translation.

Our scientists developed a novel high-specificity gene-editing endonuclease with nickase functionality that enables high-efficiency on-target single-strand breaks in genomic DNA and scarless targeted gene insertion in cells⁵. This technology can be used to improve transgene insertion, reduce off-target effects, minimize cellular toxicity, and enable enhanced safety for therapeutic applications.

The Gene-Editing Endonuclease with Nickase Functionality is protected by a pending U.S. patent (with additional patents pending in other countries).

• Ultra-high efficiency editing of primary cells and pluripotent stem cells
• Ultra-high specificity gene editing
• Virus-free and DNA-free gene editing
• Scarless gene repair using a DNA-repair template
• Donor sequence insertion into a target genomic locus (e.g., TRAC, AAVS1 safe harbor, etc.)
• Gene-editing therapies (ex vivo and in vivo)

• Autologous and allogeneic engineered cell therapies (e.g., CAR-T, CAR-NK, stem cell-derived therapies, etc.)
• Combine with Factor’s mRNA Cell Reprogramming technology to generate models of genetic disease, gene-corrected patient-specific cell therapies, and allogeneic (i.e., immuno-nonreactive or “stealth”) cell therapies, including allogeneic pluripotent stem cell-derived CAR-T and CAR-NK cell therapies for the treatment of cancer, and engineered mesenchymal stem cell (MSC) therapies for regenerative medicine, wound-healing, inflammatory and auto-immune diseases, and tumor-targeting applications