The collaboration was based on a mutual interest in forging transformative therapies for patients with rare diseases
GenKOre, Inc., a Korea-based biotech company with hyper-compact CRISPR-Cas tools, has entered into a strategic research collaboration with a US-based biopharmaceutical company to develop in vivo gene editing therapies.
This collaboration leverages GenKOre’s proprietary CRISPR Cas platform, TaRGET (Tiny nuclease, aenlarge RNA-based Genough Editen Ttechnology). The TaRGET platform differs from the most popular genome editing technology, CRISPR-Cas9, in that the entire editing module can be delivered with a single AAV vector.
Based on their TaRGET platform, GenKOre has developed several 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 the TaRGET platform in the application of in vivo gene editing therapy,” said Yongsam Kim, CEO of GenKOre. “This collaboration not only offers the opportunity to validate the applicability of the TaRGET platform in vivo therapy, but we will also extend the utility of our technologies to a broader spectrum of rare diseases.”
Under the terms of the agreement, GenKOre will receive up to $300 million, including upfront payments, option exercise fees and milestone payments upon successful achievement of R&D and commercial milestones in two in vivo disease targets. In addition, GenKOre receives research funding and tiered royalties up to a double-digit percentage of net sales.