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GenKOre, Inc., a Korea-based biotech company with hypercompact CRISPR-Cas tools, has announced it has entered into a strategic research collaboration with a U.S.-based biopharma company for the development of in vivo gene editing therapies. This collaboration will utilise 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 at 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 is built on mutual interest in forging transformative therapeutics for patients with rare diseases. Under the terms of the agreement, GenKOre will receive up to USD $300 million, including 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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