ToolGen on path to develop rare disease drugs based on patented gene editing tool

South Korea’s biotechnology company ToolGen, Inc. is on path to develop therapies to treat Charcot Marie Tooth (CMT) disease, macular degeneration, and hemophilia by utilizing its patented genome editing technology. A CMT drug is now the closest to a clinical stage among the company’s pipeline drugs.

During a recent interview with Maeil Business Newspaper, ToolGen’s chief executive Kim Young-ho said a preclinical trial of its CMT therapy will be initiated in the U.S. this year. Clinical trials will follow sequentially after the animal study that will last for about 12 months.

The company located in Seoul’s Gasan Digital Complex, the country’s biggest IT center, is a pioneer in the use of the “genetic scissors” for the development of therapies to target gene-related rare diseases. It is one of the three global players that have the cutting-edge genome editing tool, also known as CRISPR-Cas9, which is widely used in biomedical research and biotechnology.

ToolGen is the only Korean company that has CRISPR technology, and globally ToolGen has only two rivals: UC Berkeley and Broad Institute of MIT and Harvard. This is a rare area in advanced biotech fields where a Korean company can stand against global players, Kim stressed.

ToolGen recently challenged the top U.S. schools in patent interference dispute over the CRISPR technology. The U.S. Patent and Trademark Office (USPTO) initiated an interference proceeding for ToolGen. If the company wins the review, ToolGen can be granted priority as the first investor for the technology. ToolGen has one of the earliest filing dates for the use of CRISPR in mammalian systems in several countries.

The company’s early research into gene editing led by ToolGen’s co-founder Dr. Kim Jin-soo led to its high-level technology performance and global fame.

In the early 2000s, Dr. Kim developed a first-generation gene editing technology that can make a DNA-binding protein called a' gene switch' using zinc-finger protein (JFN). The development was followed by second- and third-generation CRISPR gene scissors technology completed in 2010 and 2012, respectively.

The company has also raised its global potential by moving fast in filing of patents related to the technology. The company has so far secured 33 global patents over genetic scissors.

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