On the 21st, ToolGen, a gene editing specialist company, announced that it has filed a patent infringement lawsuit against three companies: Vertex Pharmaceuticals in the United States, its partner Lonza in Switzerland, and Roslin Cell Therapies in the United Kingdom.
Earlier, ToolGen registered a patent in Europe and Japan for a method of introducing 'Cas9'—a key component of CRISPR gene editing—in protein form into cells in October last year.
CRISPR gene editing is an enzyme complex that cuts specific genes. The gene editing tool consists of guide ribonucleic acid (RNA) and Cas9 enzyme protein. When the guide RNA recognizes and binds to the DNA segment it needs to cut, the Cas9 protein attaches to the DNA and cuts it.
ToolGen's Ribonucleoprotein (RNP) is a technology that directly delivers the Cas9 protein to cells, preventing the insertion of foreign genes and reducing cellular toxicity. It is widely applied not only in plant and animal gene editing but also in drug development. The company reports that this patented technology is used in the gene therapy 'CASGEVY' developed by Vertex.
CASGEVY is a treatment for the hereditary rare diseases sickle cell disease and beta-thalassemia, and it is the first CRISPR-based gene editing therapy in the world. It was co-developed by Vertex and CRISPR Therapeutics, receiving European marketing authorization last February and starting in the UK in November 2023. The cost for a single treatment is about $2.2 million (31 billion won), and it is expected to be a blockbuster drug with annual revenues exceeding hundreds of billions of won.
ToolGen explained that this patent infringement lawsuit is not aimed at restricting the use of CASGEVY by patients in the UK, but rather is intended to obtain fair compensation for the technology owned by ToolGen through a reasonable technology contract.
Yoo Jong-sang, CEO of ToolGen, said, "CASGEVY from Vertex was born using ToolGen's CRISPR RNP technology," and added, "We expect Vertex to acknowledge the use of ToolGen's CRISPR gene editing and CRISPR RNP technology to create this innovative treatment and pay a fair price for it."