ToolGen, Inc. (KOSDAQ: 199800), a biotechnology firm at the forefront of genome editing innovations, has filed a patent infringement lawsuit in the United Kingdom against Vertex Pharmaceuticals and its key manufacturing partners, Lonza and RoslinCT. The legal action centers on Vertex’s approved CRISPR-based gene therapy, CASGEVY, which ToolGen alleges utilizes its proprietary technology without proper authorization.
ToolGen claims the development and commercialization of CASGEVY infringe upon its intellectual property related to the use of CRISPR RNP (ribonucleoprotein complex) technology. The South Korea-based company holds global patents, including those granted in Europe, for its CRISPR RNP methodology. This advanced technique enables the direct delivery of the Cas9 protein into target cells in its active protein form—bypassing the use of DNA or mRNA vectors. Such an approach not only reduces the risk of genomic integration and cellular toxicity but also offers enhanced precision and safety, making it a preferred platform in human therapeutics, as well as agricultural and animal biotechnology.
CASGEVY made history as the first gene editing therapy to be approved for clinical use, beginning with authorization by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) in November 2023, and subsequently by the European Commission in February 2024.
In December 2023, Vertex signed a licensing deal with Editas Medicine to access CRISPR-related technology. However, ToolGen asserts that this agreement does not cover critical components of the CRISPR RNP delivery system, which it pioneered and for which it holds enforceable patents.
“CASGEVY is a remarkable achievement, but it rests on a technological foundation developed by ToolGen. We believe Vertex’s therapy makes essential use of our CRISPR RNP and Cas9 technologies, and it is only fair that ToolGen be properly compensated through an appropriate licensing arrangement.”
Dr. Ryu clarified that the company’s legal move is not intended to hinder patient access to the life-changing treatment.
“This action is not about limiting access to CASGEVY in any way,” he said. “Our goal is to ensure that ToolGen’s contributions are justly recognized and rewarded.”
The lawsuit is expected to draw attention from across the biotech sector, as it highlights ongoing disputes in the fast-evolving field of genome editing, where overlapping patents and complex licensing agreements remain a point of contention.
As the legal process unfolds, industry observers will be watching closely, not only for the outcome of the case but also for its potential implications on future licensing practices and collaborations in CRISPR therapeutics worldwide.