GenEditBio has announced that it has entered into a strategic cross-license agreement with ToolGen.
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.
GenEditBio is developing GEB-200, an investigational in vivo genome-editing therapy targeting atherosclerotic cardiovascular disease (ASCVD).
The company possesses proprietary LNP and other key delivery technologies optimised for the safe and efficient in vivo delivery of genome-editing agents such as CRISPR-Cas9.
GEB-200 is designed to permanently lower plasma levels of lipoprotein(a), or Lp(a), a genetic risk factor affecting approximately 20% of the global population.
The programme has gained attention as a potential once-and-done alternative to existing chronic therapies in the cardiovascular field.
ToolGen and GenEditBio have been closely collaborating from the early development stages of GEB-200, combining their respective strengths to drive the programme forward.
The companies recently completed a successful proof-of-concept study in non-human primates (NHPs), which demonstrated effective target gene modulation and a favourable 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 jointly exploring global licencing opportunities under mutually agreed terms.
GenEditBio co-founder and Chair Prof. Zongli Zheng said: "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 the 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 added: "Investor interest in gene-editing therapies targeting cardiovascular diseases is growing rapidly."
"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."