ToolGen Initiates Patent Infringement Lawsuit Against Vertex in the UK

ToolGen's Legal Action Against Vertex Pharmaceuticals

In a significant development within the biotechnology sector, ToolGen, Inc., a prominent gene editing technology firm based in South Korea, has officially initiated a legal proceeding in the United Kingdom against Vertex Pharmaceuticals. The lawsuit specifically targets Vertex and its manufacturing partners, Lonza and RoslinCT, regarding the patent infringement related to Vertex's innovative gene editing therapy, CASGEVY.

The CEO of ToolGen, Jong Sang Ryu, announced the lawsuit on April 21, 2025, emphasizing that the move aims to protect the company's extensive portfolio of CRISPR RNP (ribonucleoprotein complex) patents, which are globally recognized, including in Europe. The patented CRISPR RNP technology enables the direct delivery of the Cas9 protein into cells, marking a revolutionary advancement in gene editing that mitigates the cellular toxicity often associated with traditional DNA and mRNA delivery systems. This technology significantly reduces off-target effects and is gaining traction in therapeutic development as well as in genomic editing of plants and animals.

CASGEVY has made history as the first gene editing therapy to receive market authorization, starting in the UK in November 2023 and subsequently gaining approval from the European Commission in February 2024. It's designed to treat patients suffering from sickle cell disease and beta-thalassemia, with the treatment pricing at approximately £1.7 million (about $2.2 million), highlighting its high potential sales forecasts. Notably, in December 2023, Vertex entered a licensing agreement with Editas Medicine for CRISPR gene editing technology, reinforcing the growing market for such innovative therapies.

In response to the lawsuit, Jong Sang Ryu stated, "Vertex's CASGEVY is derived from ToolGen's patented CRISPR RNP technology. As the pioneering company behind the first CRISPR Cas9-based gene therapy, we believe Vertex should fairly acknowledge ToolGen's contributions through a proper licensing agreement for the use of our CRISPR Cas9 and CRISPR RNP technologies in this groundbreaking treatment."

Ryu also clarified that the lawsuit is not intended to restrict patient access to CASGEVY in the UK. Instead, it emphasizes the importance of recognizing and rewarding ToolGen appropriately through an equitable licensing arrangement.

This legal clash underscores the increasing complexities of intellectual property in the fast-paced biopharmaceutical landscape, where companies are racing to develop novel therapies that leverage gene editing techniques. As more gene therapies reach the market, the importance of sound patent practices and proper licensing agreements becomes paramount to ensure fair recognition of innovations.

In essence, ToolGen's actions signal a broader call for accountability in the industry, aiming to protect its innovations while ensuring that patient access to vital therapies is maintained. The unfolding of this lawsuit will be closely watched by stakeholders within the biotech community, particularly as it could set precedents for future gene therapy developments and the intricate relationship between innovation and patent law.