ToolGen Files Patent Infringement Lawsuit Against Vertex in the United Kingdom

• ToolGen commences legal action in UK court over Vertex's genome editing therapy, CASGEVY ®
• Seeks to expand licensing of its proprietary CRISPR RNP technology

SEOUL, South Korea, April 21, 2025 /PRNewswire/ -- ToolGen, Inc. (KOSDAQ 199800), led by CEO Jong Sang Ryu and recognized as a global leader in genome editing technology, announced today that it has filed a patent infringement lawsuit in the United Kingdom against Vertex Pharmaceuticals and its commercial manufacturing partners, Lonza and RoslinCT, regarding Vertex's genome editing therapy CASGEVY.

ToolGen has secured patents for CRISPR RNP (Ribonucleoprotein complex) worldwide including in Europe. CRISPR RNP represents a groundbreaking method that delivers the Cas9 protein directly into cells in its protein form. This approach avoids the cellular toxicity often associated with DNA- and mRNA-based systems and eliminates the risk of foreign DNA integration into the genome. With its significantly lower off-target effects providing superior safety profiles, the technology is now widely adopted in therapeutic development as well as in plant and animal genome editing.

CASGEVY became the world's first genome editing therapy to receive marketing authorization, beginning with the United Kingdom in November 2023, followed by the European Commission (EC) in February 2024. It provides a novel treatment for patients with sickle cell disease and beta-thalassemia. The one-time treatment is reportedly priced at approximately £1.7 million (approx. $2.2 million USD), with sales projections indicating blockbuster potential. In December 2023, Vertex entered into a licensing agreement with Editas Medicine for CRISPR genome editing technology.

ToolGen's CEO Jong Sang Ryu commented, "Vertex's CASGEVY was born from ToolGen's proprietary CRISPR RNP technology. As the company behind the world's first CRISPR Cas9-based gene therapy, we believe Vertex should fairly recognize ToolGen's contribution through appropriate licensing for the use of our CRISPR Cas9 and CRISPR RNP technologies in developing this innovative treatment."

He continued, "This lawsuit is not aimed at restricting patient access to CASGEVY in the United Kingdom. Rather, it seeks to ensure ToolGen is properly acknowledged and rewarded through a fair licensing agreement."

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