An idea originating from a research laboratory has developed into an innovative new drug candidate, resulting in an export jackpot worth 750 billion won. This achievement is even more significant as it is the result of combining translational research by a physician-scientist (M.D.-Ph.D. Physician Scientist) with venture entrepreneurship.
On October 10, KAIST announced that SovaGen, a faculty startup founded by Professor Lee Jeongho of the Graduate School of Medical Science and Engineering (co-CEOs Park Cheolwon and Lee Jeongho), has successfully developed an 'RNA new drug candidate' for the treatment of intractable epilepsy and exported this technology to the Italian pharmaceutical company Angelini Pharma.
(From left) Professor Lee Jeongho, CEO Park Cheolwon, Senior Researcher Park Sangmin. Provided by KAIST
Previously, Professor Lee's research team was the first in the world to identify that 'acquired mutations in brain stem cells (brain somatic mutations)' are the cause of intractable epilepsy and malignant brain tumors, among other brain diseases.
The related research findings were published in Nature in 2015 and Nature Medicine in 2018, respectively.
After identifying the cause of brain diseases, Professor Lee's team, together with SovaGen CEO Park Cheolwon, an expert in new drug development, discovered an RNA drug (Antisense Oligonucleotide, ASO) that directly targets the causative mutant gene 'MTOR' for epilepsy. By successfully signing a large-scale technology transfer agreement with a global pharmaceutical company, they opened up the possibility of commercialization.
This has established a virtuous cycle in which ideas originating from basic research laboratories are developed into the world's first new drug (First-In-Class, innovative new drug) candidates through startups and advance into the global market.
Professor Lee stated, "Unlike most medical schools in Korea, which focus on patient care, KAIST has a research culture that emphasizes innovation and industrialization. This has become the driving force for simultaneously achieving two outcomes: innovative basic research and the export of new drug technologies. I expect that this achievement will serve as a good example of the future direction of KAIST's medical science research."
KAIST President Lee Kwanghyung said, "This achievement is a representative example of KAIST's research philosophy (from basic research to industry) being realized in the field of medical science. KAIST will continue to lead innovation for human health and the future bioindustry through challenging basic research."
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