GenEdit aims to expand with gene therapy

GenEdit Chief Executive Officer and Co-Founder Lee Kun-woo

GenEdit Inc., a genetic pharmaceutical firm based in Silicon Valley, the United States, is looking to collaborate with leading companies in the biotech industry, Chief Executive Officer and Co-Founder Lee Kun-woo said.

GenEdit, a genetic medicine startup founded by South Koreans in the United States, recently gained the industry’s attention after it announced a collaboration and license agreement with Genentech, a member of the Roche Group. Under the agreement, GenEdit will receive up to $629 million in preclinical and clinical development, commercial, and net sales milestone payments, according to the company’s announcement.

Genentech is also headquartered in South San Francisco. The city, which was once filled with warehouses, has now become one of the world’s largest innovation hubs for biotech since the company was founded in 1976.

“I consider it a great honor to collaborate with Genentech, which I have admired in the past,” Lee said, in an interview with Maeil Business Newspaper. “I want this collaboration to become a success story for Korean biotech companies.”

GenEdit aims to achieve significant results in the biotech field, following in the footsteps of successful startups founded by Korean entrepreneurs in Silicon Valley.

GenEdit was founded as a startup in 2016 by Lee and Park Hyo-min, senior vice president of business operations, GenEdit in UC Berkeley. GenEdit established a corporation in Korea in 2022 and has been hiring Korean researchers, with Park heading this Korean entity.

The company developed the NanoGalaxy platform, a soluble nano-particle delivery system that can efficiently transport gene therapy substances to the necessary parts of the patient’s body. Under the agreement, GenEdit will receive an upfront payment of $15 million from Genentech and is eligible for technology fees at each stage, including research, preclinical, clinical, and commercialization successes. If the therapy is commercialized, GenEdit can also receive separate royalties based on sales.

The two companies will collaborate to develop gene therapies for autoimmune diseases based on GenEdit’s NanoGalaxy platform. GenEdit will work on developing soluble nanoparticles for autoimmune diseases in collaboration with Genentech, while Genentech will be responsible for preclinical and clinical development, clinical approval, and commercialization.

“Genentech was innovative by becoming the first company to create protein-based drugs,” Lee said. “Protein-based drugs have now become the majority of blockbuster drugs in the pharmaceutical market after 30 years.”

The next 30 years will be centered around gene-based drugs, and it has already become a reality with the emergence of mRNA vaccines for Covid using mRNA technology, he added.

“Until now, autoimmune diseases were treated with therapies that suppress the immune reaction itself,” Lee said. “As our understanding of the immune system increases, there is a growing expectation that, just as we can command the immune cells to attack with increased knowledge, we can also suppress specific immune reactions by commanding them not to attack.”

The NanoGalaxy Platform created by GenEdit aims to deliver any gene therapy substance to the target organ. When this becomes possible, patients can be effectively treated with much cheaper gene therapies.

The technology also demonstrates the potential for gene correction, as shown in the research on CRISPR gene scissors conducted Lee alongside Nobel laureate and UC Berkeley Professor Jennifer Doudna.

“The U.S. FDA approved the first CRISPR gene scissors therapy, Casgevy, in December 2023, but the cost is as high as $2.2 million per application,” Lee said. “The goal is to make this technology available to more people.”

GenEdit has received investment from Eli Lilly, currently one of the world’s largest pharmaceutical companies. Eli Lilly fosters biotech companies in its South San Francisco office, where GenEdit’s office is located.

In addition to Eli Lilly, well-known Silicon Valley venture capitalists including Sequoia Capital LLP, Bow Capital, and DCVC Bio, as well as Korean venture capital firms, have participated as investors.