Adenine base editors were successfully created by GenKOre, a biotech startup in South Korea, using its unique hypercompact gene-editing method.
The hypercompact ABEs created by GenKOre are distinguished by their small size, ability to be delivered via an adeno-associated virus (AAV), and base-editing activity that is both selective and flexible and has been verified in vivo. Previous base-editing technology was created using a SpCas9 nuclease that is so large that it cannot be carried by AAV vectors that have a payload cap, and is therefore restricted to hepatic distribution. Through AAV delivery, this new base-editing method is anticipated to offer a range of in vivo therapy options for genetic illnesses.
To recruit a vast pool of talent for the creation of hypercompact ABEs-based gene therapy, GenKOre recently increased the size of its research facility in Seoul. The company is also looking for partnerships with academic institutions and pharmaceutical firms. Additionally, it is growing its ex vivo gene therapy business through strategic alliances with organisations that have a focus on immune cells and hematopoietic stem cells. Thus, ex vivo gene-editing treatments will be developed using the ABE platform.