SEOUL, South Korea, Jan. 26, 2023 /PRNewswire/ -- GenKOre, Inc., a Korea-based biotech company with hypercompact  CRISPR-Cas tools,  announced that it has  entered into  a  strategic research collaboration with a US-based  biopharma  company for the development of  in vivo  gene  editing  therapies. This  collaboration will utilize  GenKOre's  proprietary  CRISPR-Cas  platform,  TaRGET  (Tiny nuclease,  augment  RNA-based  Genome  Editing  Technology). The  TaRGET  platform is distinct from the most popular  genome editing technology,  CRISPR-Cas9,  in that the whole editing module can be delivered with a single AAV vector. Based on  their  TaRGET platform,  GenKOre  has developed different  modalities  of editing tools including  TaRGET-CUT,  TaRGET-Adenine Base Editing (ABE),  TaRGET-AI (Gene Activation and Inhibition system).  and  TaRGET-FREE  (Gene knock-in)  .  

"This collaboration reflects the strength and  potential  utility of  TaRGET  platform in the application of  in vivo  gene-editing therapy", said Yong-Sam Kim, CEO  of  GenKOre. "Not only will this collaboration provide an opportunity to validate the applicability of  TaRGET  platform to  in vivo  therapy, but we will also expand the utility of our technologies to a wider spectrum of rare diseases." The collaboration  was  built on the mutual  interest  in  forging transformative therapeutics for patients with rare diseases.  

Under the terms of the agreement,  GenKOre  will receive  up to  300 million USD including an upfront, option exercise fees and milestone payments upon successful achievement of  R&D  and commercial milestones across two  in vivo  disease targets. In addition,  GenKOre  will receive research funding and tiered royalties up to a double-digit percentage of net sales.  

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