GenKOre, a biotech startup in South Korea, succeeded in the development of adenine base editors (ABEs) based on its own hypercompact gene-editing technology. 

The hypercompact ABEs developed by GenKOre is characterized by compactness in size that can be delivered using adeno-associated virus (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.