ToolGen and GenEditBio Unite for Cutting-Edge Genome-Editing Therapy Advancement
In a groundbreaking move to enhance the development of genome-editing therapeutics, ToolGen Inc., based in Korea, has entered a strategic cross-license agreement with GenEditBio Limited, a biotechnology firm located in Hong Kong. This partnership unites two pioneering technologies: ToolGen's renowned CRISPR-Cas9 genome editing platform and GenEditBio's advanced lipid nanoparticle (LNP) delivery system. Together, they aim to foster the development of innovative in vivo genome-editing therapeutics that promise transformative treatment options for diseases with significant unmet medical needs.
This collaboration leverages the strengths of both companies, enhancing the precision and delivery of genome editing therapies. The CRISPR-Cas9 technology, recognized for its ability to make high-precision edits in genetic material, serves as the foundation, while GenEditBio's expertise in LNP delivery systems ensures the safe and effective transport of therapeutic cargo to targeted tissues, crucial for successful in vivo editing.
As part of this collaboration, the focus will be on GEB-200, an investigational therapy for atherosclerotic cardiovascular disease (ASCVD). GenEditBio has developed this therapy to target lipoprotein(a), a genetic risk factor present in roughly 20% of the global population. By lowering Lp(a) levels permanently, GEB-200 has emerged as a viable contender against existing chronic treatments in the cardiovascular sector, embodying the “once-and-done” treatment paradigm.
The partnership is already yielding promising results. ToolGen and GenEditBio have collaborated closely from GEB-200's early development phases, culminating in a successful proof-of-concept study conducted in non-human primates (NHPs). This study demonstrated effective gene modulation and an acceptable safety profile, which represents a critical milestone toward submitting an Investigational New Drug (IND) application.
Moving forward, the two companies plan to expedite GEB-200’s regulatory journey by utilizing their combined capabilities. They also aim to pursue global licensing opportunities on mutually beneficial terms. This strategic collaboration aligns with a broader industry trend toward innovative gene-editing solutions, as highlighted by recent investments in gene therapy targeting cardiovascular diseases. Notably, Eli Lilly's $1 billion acquisition of Verve Therapeutics exemplifies the rising investor interest in this area and signifies the potential market impact for successful therapies such as GEB-200.
Prof. Zongli Zheng, Co-founder and Chairman of GenEditBio, emphasized their shared philosophy: “Together, We Are Stronger.” He expressed excitement about the collaboration, focusing on the potential to provide safe and effective genome-editing therapies to patients. Meanwhile, Jong Sang Ryu, CEO of ToolGen, noted the increasing interest in gene-editing therapies aimed at cardiovascular ailments, underscoring their goal of delivering revolutionary treatments by merging their CRISPR expertise with GenEditBio’s state-of-the-art delivery systems.
About ToolGen: Founded in Korea, ToolGen Inc. stands as a prominent figure in biotechnology, particularly known for its pioneering patent portfolio surrounding CRISPR-Cas9. The company actively engages in research and development in three core areas: Intellectual Property Monetization, Therapeutics, and Crop Science. With its comprehensive patented technology, ToolGen affirms its leadership in delivering precise genetic modifications applicable across various biological fields.
About GenEditBio: Established in 2021, GenEditBio operates with the mission to offer innovative and potentially curative therapies utilizing in vivo genome editing. Focusing on novel Cas nuclease discoveries and safe delivery methods, the company aims to create programmable genetic therapies that ensure patient safety, precision, and affordability, marking significant advancements in treating genetic diseases. With backing from several leading life science investors, GenEditBio continues to carve out a niche in biotechnology, dedicating substantial efforts to transformative healthcare solutions.
This collaboration leverages the strengths of both companies, enhancing the precision and delivery of genome editing therapies. The CRISPR-Cas9 technology, recognized for its ability to make high-precision edits in genetic material, serves as the foundation, while GenEditBio's expertise in LNP delivery systems ensures the safe and effective transport of therapeutic cargo to targeted tissues, crucial for successful in vivo editing.
As part of this collaboration, the focus will be on GEB-200, an investigational therapy for atherosclerotic cardiovascular disease (ASCVD). GenEditBio has developed this therapy to target lipoprotein(a), a genetic risk factor present in roughly 20% of the global population. By lowering Lp(a) levels permanently, GEB-200 has emerged as a viable contender against existing chronic treatments in the cardiovascular sector, embodying the “once-and-done” treatment paradigm.
The partnership is already yielding promising results. ToolGen and GenEditBio have collaborated closely from GEB-200's early development phases, culminating in a successful proof-of-concept study conducted in non-human primates (NHPs). This study demonstrated effective gene modulation and an acceptable safety profile, which represents a critical milestone toward submitting an Investigational New Drug (IND) application.
Moving forward, the two companies plan to expedite GEB-200’s regulatory journey by utilizing their combined capabilities. They also aim to pursue global licensing opportunities on mutually beneficial terms. This strategic collaboration aligns with a broader industry trend toward innovative gene-editing solutions, as highlighted by recent investments in gene therapy targeting cardiovascular diseases. Notably, Eli Lilly's $1 billion acquisition of Verve Therapeutics exemplifies the rising investor interest in this area and signifies the potential market impact for successful therapies such as GEB-200.
Prof. Zongli Zheng, Co-founder and Chairman of GenEditBio, emphasized their shared philosophy: “Together, We Are Stronger.” He expressed excitement about the collaboration, focusing on the potential to provide safe and effective genome-editing therapies to patients. Meanwhile, Jong Sang Ryu, CEO of ToolGen, noted the increasing interest in gene-editing therapies aimed at cardiovascular ailments, underscoring their goal of delivering revolutionary treatments by merging their CRISPR expertise with GenEditBio’s state-of-the-art delivery systems.
About ToolGen: Founded in Korea, ToolGen Inc. stands as a prominent figure in biotechnology, particularly known for its pioneering patent portfolio surrounding CRISPR-Cas9. The company actively engages in research and development in three core areas: Intellectual Property Monetization, Therapeutics, and Crop Science. With its comprehensive patented technology, ToolGen affirms its leadership in delivering precise genetic modifications applicable across various biological fields.
About GenEditBio: Established in 2021, GenEditBio operates with the mission to offer innovative and potentially curative therapies utilizing in vivo genome editing. Focusing on novel Cas nuclease discoveries and safe delivery methods, the company aims to create programmable genetic therapies that ensure patient safety, precision, and affordability, marking significant advancements in treating genetic diseases. With backing from several leading life science investors, GenEditBio continues to carve out a niche in biotechnology, dedicating substantial efforts to transformative healthcare solutions.
Topics Health）
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