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KAIST Exports Global License for New Drug Candidate for Intractable Epilepsy Worth 750 Billion KRW
<(From Left) Professor Jeong Ho Lee, CEO Cheolwon Park, Principal Researcher Sang-min Park>
KAIST (President Kwang Hyung Lee) announced on the 9th of October that Sovargen (co-led by CEOs Cheolwon Park and Jeong Ho Lee), a faculty startup led by Professor Jeong Ho Lee of the KAIST Graduate School of Medical Science and Engineering, has successfully achieved a global technology export deal worth a total of 750 billion KRW. The deal involves an innovative RNA-based new drug candidate for the treatment of intractable epilepsy.
This achievement is drawing attention as a representative example of how groundbreaking discoveries from KAIST’s fundamental medical science research can evolve into actual drug development and global market expansion.
Professor Jeong Ho Lee’s research team was the first in the world to identify that the cause of severe brain diseases such as intractable epilepsy and malignant brain tumors lies in brain somatic mutations—acquired mutations that occur in neural stem cells. Their findings were published in Nature (2015) and Nature Medicine (2018).
Later, together with Cheolwon Park of Sovargen, an expert in drug development, they discovered an RNA-based therapeutic—an Antisense Oligonucleotide (ASO)—that directly targets MTOR, a key mutated gene responsible for epilepsy. Through a large-scale technology transfer agreement with a global pharmaceutical company, they also demonstrated the drug’s commercial potential.
This achievement is particularly significant in that it was led by Professor Jeong Ho Lee, a physician-scientist (M.D.-Ph.D.) who integrates intensive basic research with translational studies and venture entrepreneurship.
An idea that originated in a basic research lab has developed into the world’s first innovative drug (first-in-class) candidate through a startup, creating a virtuous cycle that connects back to the global market.
Sovargen’s Principal Researcher Sang Min Park (KAIST Graduate School of Medical Science and Engineering alumnus) stated, “From identifying the disease cause to developing a new drug and exporting the technology globally, this achievement was made possible entirely through the power of Korean science.” Sovargen CEO Cheolwon Park added, “This success was made possible thanks to the strong support of President Kwang Hyung Lee and key KAIST leaders for both the Graduate School of Medical Science and Engineering and faculty-led startups.”
Professor Jeong Ho Lee commented, “While traditional medical schools in Korea are centered around clinical practice, KAIST fosters a research culture focused on innovation and industrialization. This enabled us to achieve both groundbreaking basic research and global new drug technology export.” He continued, “This success serves as an excellent example of the future direction of KAIST’s medical science research.”
Experts have evaluated this accomplishment as one that opens new therapeutic possibilities for patients suffering from intractable epilepsy—conditions that previously had no treatment options—while also demonstrating that Korean medical science and biotech ventures are capable of competing on the global stage in innovative new drug development.
KAIST President Kwang Hyung Lee remarked, “This achievement is a representative example of how KAIST’s research philosophy—‘from fundamentals to industry’—has been realized in the field of medical science.” He added, “KAIST will continue to pursue bold fundamental research to lead innovations that advance human health and the future bioindustry.”
2025.10.10 View 1511 -
Professor J.H. Lee Wins the Innovators in Science Award
Professor Jeong Ho Lee from the Graduate School of Medical Science and Engineering won the Early-Career Scientist Award of the 2020 Innovators in Science Award. The New York Academy of Sciences administers the award in partnership with Takeda Pharmaceutical Company.
The Innovators in Science Award grants two prizes of US $200,000 each year: one to an Early-Career Scientist and the other to a well-established Senior Scientist who have distinguished themselves for the creative thinking and impact of their rare disease research. The Senior Scientist Awardee is Dr. Adrian R. Krainer, at Cold Spring Harbor Laboratory whose research focused on the mechanisms and control of RNA splicing.
Prof. Lee is recognized for his research investigating genetic mutations in stem cells in the brain that result in rare developmental brain disorders. He was the first to identify the causes of intractable epilepsies and has identified the genes responsible for several developmental brain disorders, including focal cortical dysplasia, Joubert syndrome—a disorder characterized by an underdevelopment of the brainstem—and hemimegaloencephaly, which is the abnormal enlargement of one side of the brain.
“It is a great honor to be recognized by a jury of such globally respected scientists whom I greatly admire,” said Prof. Lee. “More importantly, this award validates research into brain somatic mutations as an important area of exploration to help patients suffering from devastating and untreatable neurological disorders.”
Prof. Lee also is the Director of the National Creative Research Initiative Center for Brain Somatic Mutations, and Co-founder and Chief Technology Officer of SoVarGen, a biopharmaceutical company aiming to discover novel therapeutics and diagnosis for intractable central nervous system (CNS) diseases caused by low-level somatic mutation.
The Innovators in Science Award is a limited submission competition in which research universities, academic institutions, government or non-profit institutions, or equivalent from around the globe with a well-established record of scientific excellence are invited to nominate their most promising Early-Career Scientists and their most outstanding Senior Scientists working in one of four selected therapeutic fields of neuroscience, gastroenterology, oncology, and regenerative medicine. The 2020 Winners will be honored at the virtual Innovators in Science Award Ceremony and Symposium in October 2020.
2020.07.09 View 14607