AAV-loadable hypercompact ABEs developed by GenKOre

DAEJEON, South Korea, Aug. 2, 2022 /PRNewswire/ -- GenKOre, a biotech start-up in Korea is a game changer in gene-editing therapy area with its own hypercompact gene-editing technology. The company also successfully developed hypercompact adenine base editors (ABEs), which were introduced in Nature Chemical Biology on August 2, 2022.

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 cannot be delivered by payload-limiting AAV vectors and has been thus confined for hepatic delivery using lipid nanoparticles. But these new hypercompact ABEs can be loaded into a single AAV vector and boast of high base editing efficiency and specificity. The company also validated base-editing feasibility in mouse in vivo.

GenKOre (homepage: www.genkore.com) is a biotech 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 highly efficient genome editing platform. They develop AAV/non-viral-based gene therapy and new plant varieties using its own genome editing technology. 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.

This new base-editing technology is expected to provide various in vivo treatment options for genetic diseases through AAV delivery. Alternatively, the ABE system is expected to harness the development of ex vivo gene-editing therapy targeting immune cells and hematopoietic stem cells.

The related article published on Nature Chemical Biology can be checked at: https://www.nature.com/articles/s41589-022-01077-5.

GenKOre website: www.genkore.com

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