GenKOre develops hypercompact adenine base editors (ABEs)

02 August 2022 | Tuesday | News

GenKOre, a biotech start-up in Korea, succeeded in the development of ABEs based on its own hypercompact gene-editing technology and had the relevant study published on Nature Chemical Biology on August 2, 2022.

GenKOre (homepage: is a business spun off from the Korea Research Institute of Bioscience and Biotechnology (KRIBB), which is a government-invested research institute in Korea. It has platform business through proprietary gene-editing platform. GenKOre succeeded in the development of a hypercompact CRISPR system with AAV delivery compatibility termed TaRGET (Tiny nuclease/augment RNA-based Genome Editing Technology), which was published on Nature Biotechnology in 2021. The AAV-loadable ABE technology was derived from the TaRGET platform. 

The hypercompact ABEs developed by GenKOre is characterized by compactness in size that can be delivered using AAV and boast of specific and versatile base-editing activity, which was also validated in vivo. Previous base-editing technology was developed based on such a 'big' SpCas9 nuclease that it cannot be delivered by payload-limiting AAV vectors and has been thus confined for hepatic delivery. This new base-editing technology is expected to provide various in vivo treatment options for genetic diseases through AAV delivery.

GenKOre recently expanded its research facility in Seoul in an effort to attract a large number of human resources for the development of hypercompact ABEs-based gene therapy, seeking collaborations with pharmaceutical companies and academia. Besides, it is expanding business to ex vivo gene therapy by making strategic alliance with companies with expertise in immune cells and hematopoietic stem cells. The ABE platform will be thus employed to develop ex vivo gene-editing therapy. 


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