[CEO Interview] Attracted billions of investment by developing a gene-based target treatment system for incurable diseases

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Meet Lee Seong-wook, a professor of Department of Biotechnology and Bioengineering and CEO of Rznomics, who stands out as the CEO of a promising biopharmaceutical company from an aspiring researcher

When I went to study in the United States, my advisor had already finished research on the viral replication process and started researching gene therapy using viruses as vector. Participating in these studies, he decided to pursue research in the field of intractable treatment. In addition, my advisor taught me to always think about how to develop value in the research in the laboratory, and he showed the realization of that value through starting a business.

By maximizing my research in incurable diseases such as cancer can be treated with minimal damage to normal tissues, and I believed it would be of sufficient value. And in order to realize the value, from the beginning of R&D I had the mindset that 'start-up is the way to go'. Maybe it was because I got insight from the professors who started a business, and also I already have a student who started a business. After he finished master’s degree in molecular biology, he went to medical school to become a research doctor, but after becoming a medical professor, one day, he came to me with a business card stating CEO.”

Professor Seong-wook Lee (Department of Biotechnology and Bioengineering, Graduate School) from Dankook University obtained a bachelor’s degree in microbiology from Seoul National University and a doctorate in molecular biology from Cornell University, USA which he also worked as a postdoctoral researcher. He who is also the former president of the Korean Society for Gene and Cell Therapy, compiled the results of his 20 years of research and founded Rznomics.

Rznomics is developing an RNA-based gene therapy using the trans-splicing ribozyme platform technology. This technology is a platform technology that recognizes the target site and removes the problematic RNA sequence from this site, and at the same time connects the RNA that delivers or injects a specific substance to the target site for the purpose of improving the function of the target. As the excellence of the technology is spreading widely, Rznomics is attracting large amounts of investment every year, and plans to list it on the stock market in the future.

On January 4th, I met Professor Seong-wook Lee and was able to hear detailed story about the prospects and goals of Rznomics and the technology.


Q. Congratulations on becoming a CEO who is attracting attention not only in academia but also in the biopharmaceutical industry.

All I've done is try to make sure that valuable technology doe not go dead. What kind of technology is that? It is a technology that was developed through sweat of researchers with the support of tax money collected by the government to help cure someone. Although it has received attention within Korea, Rznomics' goal is to provide licenses to global biopharmaceutical companies. If you have commercialized the technology that you have researched for decades, I think you should aim for it to growth on a global scale. There is still much work to be done to achieve that goal.


Q. Could you please explain the trans-splicing ribozyme platform technology that you have been researching for many years?

The key is to inject a ribozyme into a patient in the form of a DNA vector that removes bad RNA that causes incurable diseases and substitute it with a good RNA. Ribonucleic acids that function as enzymes are called ribozymes. Although there are various types of ribozymes, the ribozyme we have focused on researching and developing conceptually has the ability to selectively induce the activity of therapeutic genes in cells expressing therapeutic RNA by editing the target RNA to be treated with therapeutic RNA. The technology we've been researching is to take advantage of these functions of ribozymes, and our lab has for 20 years been focused on optimizing these functions beyond concept to actually have therapeutic effects. Previous studies have had marginal efficacy or safety concerns.


I have achieved the result of developing a ribozyme with therapeutic function in vivo for the first time by making the therapeutic gene RNA to be expressed only in target diseased cells (specificity), increasing therapeutic efficacy, and at the same time improving the safety of the DNA vector.



Q. Why did you choose liver cancer as a target disease for treatment development?

Cancer is still one of the most incurable diseases. In the early 2000s, when there was very little public interest in gene therapy, hepato-cancer specialists and researchers were the people who proposed research after hearing about my research field ‘trans-splicing enzyme platform’. As a result of working together with these people and immersing ourselves in the research, the liver cancer-based treatment material optimization was completed. Since it is a target molecular substance for treatment, not a drug composed of chemicals, which it is a platform technology, it has sufficient potential to be applied to all cancers.

Currently, research is being conducted to expand indications to glioblastoma of malignant brain tumors and hereditary retinal pigment degeneration. We are also investigating the possibility of using it as a treatment for neurological/cognitive diseases such as Alzheimer's and Rett's syndrome. In particular, the innovative new drug lead material for Rett Syndrome, a hereditary neurodevelopmental disorder that has no underlying treatment, was developed by Rznomics and was recently selected as a governmental new drug development project. 


Q. Based on this technology, you founded Rznomics, as a subsidiary of Dankook University Industry-University Cooperation Foundation.

Rznomics was founded in August 2017 as a research company that develops gene therapy products using RNA substitution enzymes. We are researching with the goal of developing bio-drugs for cancer/incurable diseases through gene therapy development. In particular, we are researching a treatment for primary liver cancer, and the results of the research that specifically kills cancer cells based on the 'trans-conjugated ribozyme' platform for RNA replacement enzymes were published in the official journal (Molecualr Therapy Nucleic Acids) of the American Society of Gene and Cell Therapy (ASGCT). We have applied for clinical approval to the Korean Ministry of Food and Drug Safety at the end of November last year for pipeline 'RZ-001' to which this technology has been applied targeting liver cancer treatment. As it is a field that I have researched for 20 years and achieved a high level of optimization, I decided that there was a certain possibility of commercialization, and I was able to start a business without hesitation because gene therapy is a field where investment is made actively.


Q. It is a field that is meaningful enough for pure research, but I am curious as to why you decided to start a business.


When I was doing my PhD in Molecular Biology at Cornell University, my advisor was passionate about starting a business. 'Developing skills and writing a thesis is important, but you shouldn't be content with just leaving your name on a few pages of paper. “It would be even better if we could study the application method that would actually affect society to the end and start a company based on it.” It was a time when the biopharmaceutical industry was revitalized in the United States, and I was aware from an early age that my research could be commercialized based on the latest research results such as the concept of ribozyme. Eventually, I started a company when the business plan was materialized for my research on gene therapy to be injected in the form of a vector based on RNA.


Gene therapy was introduced as a concept in the 1990s, but it was said that it was impossible to commercialize it. In the 2010s, developed countries’ authorities began to approve gene therapy. Since I started my business in 2017, so I guess I tend to adapt well to the global trend.


Q. Did you have any difficulties in the early days of going out of the lab and running the company yourself? 

Since it was a company that started with two students and a school laboratory, of course, in the beginning, we did not have a lot of money. But we didn't worry too much because we had certainty in commercializing our research. In 2017,  we was selected for a private-led start-up support project (TIPS) to raise funds for establishment, and with some help from venture capitals, we were able to receive funds as well as conducting research projects from government agencies. The research results have been attracting attention every year, and so far we have been able to attract a total of 23.7 billion KRW of investment. We had difficulties in management, administration, and legal affairs, but thanks to Dankook University Industry-University Cooperation Foundation we were provided a lot of help from early stage of the company in various areas. I would like to extend my gratitude for the support. 


Q. What does it mean for a company started with technology developed in the laboratory to receive tens of billions of investment?

When a science and engineering researcher actually start a business, establishes a company, and receives investment, researchers often joke with each other, saying, “I have crossed a river of no return.” It is because there is no guarantee that you will be successful just because you receive an investment, and you cannot be pushed back just because there is no guarantee of success. However, I have had experience of starting a small business before, and from this experience, I personally decided that in order to succeed as a biopharmaceutical company, I had to build a unique platform technology and a pipeline that can prove the value of these technologies. Rznomics was founded by trusting our technology as the preparation process has matured enough. A researcher starts a business because he thinks his research is great, but an investor makes an objective judgment and evaluation, so receiving an investment is meaningful. Investors invest in us because they believe in our company, and the amount of investment reflects our evaluation.


Q. Established as a research institute on campus, the company has grown so rapidly that it has received tens of billions of investment over the past four years. You said that you express your special thanks to the Dankook Industry-University Cooperation Foundation, and I am curious as to why.

In the early days of the founding, all members of the company were researchers. In that situation, you need to distinguish between what you can do on your own and what you must get help from. One of them is public relations and external cooperation such as PR, IR, and website production. The Industry-University Cooperation Foundation, together with the school headquarters, provided support and provided PR expenses. In addition to the BRIDGE+ commercialization development cost, Dankook University's technology holding company also made a direct equity investment in Rznomics. I express my gratitude once again for sharing our achievements with professors and other school members at important events.


Q. From the perspective of managing a promising biopharmaceutical company, would you give advice to researchers who are preparing for technology commercialization based on university research?

I too am still in a starting position, so providing 'advice' would not be the correct wording. If I dare say, I would like to point out that it is necessary to diagnose the technology soberly and accurately, and to respect the area of expertise as much as possible. I recommend that you first get an objective evaluation of your skills. All the professors are the highest authority in their field, so I think that the technology developed by them is also the best technology. However, unlike academic research, commercialization must directly generate profits. To do this, you need to take a good look at the industry, see who your competitors are in Korea and in the world, and think about where your company will stand. If I'm the first, I need to review why others haven't jumped into it, make sure it's not in an area that's unrelated to monetization, and there's no catastrophic risk. If I am the best, I need to diagnose where the technology can be applied and what the revenue will be if it is applied.


In addition, although a professor may have authority in research or technology development, but he at the same time can be unfamiliar with technology research, development for commercialization of actual technology, and other fields. As the pace of technological development is fast, the first or best technology is good, but it must be made into a company that has built an efficient system that can successfully commercialize it with the agility to present the optimal technology for the purpose at the right time. To do this, you need to open your mind to recruiting experts in each field or to collaborate with them. I want you to keep in mind that investment includes not only R&D expenses, but also personnel expenses and external activity expenses.