- Preclinical validation of a targeted treatment candidate for cardiovascular disease
- Accelerating clinical advancement through integrated gene correction and delivery technologies
[by Sung, Jae Jun] ToolGen, a gene-editing technology developer, announced on November 24 that it has entered into a strategic cross-licensing agreement with GenEditBio, a Hong Kong-based lipid nanoparticle (LNP) platform company, to co-develop in vivo gene editing therapies.
The collaboration forms part of ToolGen's open-innovation strategy and seeks to integrate the company’s CRISPR-Cas9 gene-editing technology with GenEditBio's in vivo delivery platform to develop next-generation gene-editing therapeutics with the goal of achieving a single-dose cure.
A fundamental requirement for gene-editing therapeutics is the ability to achieve precise gene modification together with efficient in vivo delivery, underscoring the importance of the technological synergy between the two companies. GenEditBio holds LNP-based delivery capabilities and is developing ‘GEB-200,’ a gene-editing candidate designed to permanently lower Lp(a) protein levels in high-risk patients with atherosclerotic cardiovascular disease (ASCVD). With an estimated 20% of the global population considered to be at elevated risk, the potential for a single-dose treatment is drawing considerable attention.
The two companies have advanced preclinical research on GEB-200 through preliminary joint studies and recently achieved successful proof-of-concept (PoC) results in non-human primates (NHP), thereby establishing a foundation for clinical development. Moving forward, both companies plan to expedite IND approval by integrating CRISPR gene-editing technology with the LNP delivery platform and intend to pursue a joint technology transfer (L/O) model for sharing profits.
"This cross-licensing agreement is expected to accelerate the development of GEB-200," GeneEditBio stated.
"Market expectations for gene-editing and cardiovascular disease therapeutics continue to rise, as evidenced by Eli Lilly’s recent acquisition of Verve Therapeutics, whose modality pipeline is comparable to GEB-200, for up to USD 1.3 billion," said Ryu Jong-sang, CEO of ToolGen.
"Through this cross-licensing agreement and co-development project with GeneEditBio, we aim to further accelerate the development of innovative gene-editing therapies and work to deliver optimal treatment options to a broader patient population," Ryu further remarked.