GenEditBio Limited ("GenEditBio"), a Hong Kong-headquartered clinical-stage biotechnology company specializing in novel deliveries, including lipid nanoparticle (LNP)-based delivery systems, announced that it has entered into a strategic cross-license agreement with ToolGen Inc., a leading genome editing company based in Korea. Through this agreement, the companies will jointly advance the development of next-generation in vivo genome-editing therapeutics.
This collaboration brings together two essential and highly complementary technologies—ToolGen's best-in-class CRISPR-Cas9 genome editing platform and GenEditBio's industry-leading LNP delivery system. Together, these platforms enable a transformative approach to genome-editing therapeutics, aiming for a single-administration ("once-and-done") treatment for diseases with significant unmet clinical needs.
ToolGen and GenEditBio have been closely collaborating from the early development stages of GEB-200, combining their respective strengths to drive the program forward. The companies recently completed a successful proof-of-concept study in non-human primates (NHPs), which demonstrated effective target gene modulation and a favorable safety profile—marking a key milestone toward IND submission.
Looking ahead, ToolGen and GenEditBio plan to accelerate the regulatory path for GEB-200 by leveraging their integrated platform capabilities and to jointly explore global licensing opportunities under mutually agreed terms.
GenEditBio Co-founder and Chairman Prof. Zongli Zheng commented:
"GenEditBio and ToolGen share the same philosophy that 'Together, We Are Stronger' and a common goal of bringing safe and effective genome-editing therapies to patients with unmet medical needs. We are excited to enter this cross-license agreement with ToolGen to accelerate the development of GEB-200 beyond preclinical stage. The collaboration attests to our high-throughput LNP barcoding system that is highly predictive in clinical translation and ToolGen's advanced CRISPR-Cas9 genome-editing technology."
ToolGen CEO Dr. Jong Sang Ryu commented:
"Investor interest in gene-editing therapies targeting cardiovascular diseases is growing rapidly. Eli Lilly's recent acquisition of Verve Therapeutics—whose pipeline includes an Lp(a)-targeted gene-editing program—for $1 billion upfront and up to $300 million in contingent value rights clearly reflects this trend. Through our strategic collaboration with GenEditBio, we aim to deliver a breakthrough in gene-editing treatment by combining ToolGen's CRISPR expertise with GenEditBio's advanced delivery technology, ultimately bringing transformative, once-and-done therapeutic options to patients worldwide."