GenKOre (homepage: www.genkore.com), a biotech start-up in Korea, had a study on the development of hypercompact adenine base editors (ABEs) published on Nature Chemical Biology on August 2, 2022.
The hypercompact ABEs newly developed by GenKOre feature a hypercompact size that can be delivered using a single AAV vector and boast of high base editing efficiency and specificity. The company also validated base-editing feasibility in mouse in vivo.
Previously, GenKOre developed a hypercompact CRISPR system with AAV delivery compatibility, coined TaRGET (Tiny nuclease/augment RNA-based Genome Editing Technology), which was published in Nature Biotechnology in 2021.
Adeno-associated virus (AAV) is one of the most validated delivery vehicles for gene therapy, but has a limited payload size to 4.7 kb. 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. The hypercompact base editing system developed by GenKOre was developed based on TaRGET system and is amenable to AAV delivery. This new base-editing technology is expected to provide various in vivo treatment options for genetic diseases through AAV delivery.
GenKOre is a start-up 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 hypercompact genome editing platform. GenKOre’s develops AAV/ non-AAV-based gene therapy and animals/plants using its own genome editing system.
Recently, GenKOre expanded its research facility in Seoul to attract more researchers and aim to develop a gene therapy based on hypercompact genome editing technology and to seek collaborations with pharmaceutical companies and academia.