Key Technology

Traditional gene editors are nucleases that can only be used when K/O is the target. This is because they produce DSBs, which result in near-random outcomes, including random insertions and deletions. They are also inefficient, particularly in non-dividing cells, and are therefore unsuitable for industrial use. We have developed a base editor that can precisely substitute only the desired bases without causing DSBs. It is highly efficient and can be used universally, including in non-dividing cells.

Article

High-efficiency base editing for nuclear and mitochondrial DNA with an optimized DYW-like deaminase.
J Kweon et al. Molecular Therapy, August, 2025 (DOI: 10.1016/j.ymthe.2025.08.007)

• A novel CRISPR-based cytosine base editor(SsCBE) using a toxin-derived DYW-like deaminase from Pseudomonas syringae (SsdAtox).

• Through rational engineering, the optimized SsCBE exhibited comparable levels of activity and reduced off-target effects relative to BE4max.

• The SsCBE was efficiently delivered by diverse methods, including adeno-associated viruses(AAVs), ribonucleoprotein (RNP) complexes, and engineered virus like particles (eVLPs).

• The versatility of SsCBE was also demonstrated through successful application of microinjection into mouse zygotes for targeted cytosine base editing.

• The utility of SsCBE was further extended to mitochondrial DNA editing through TALE array fusion

Patents

We have filed three Korean patents and PCTs. One of these has been filed for entry into individual countries, including the US, Europe, China and Japan, and is expected to be granted this year.