- [DP Interview] Nam Jae-hwan, CEO of SML Biopharm
- Secured high-stability mRNA design and AI-based LNP platform, targeting infectious diseases and cancer
- “Accelerating development of an SFTS therapeutic based on our proprietary platform… strengths in versatility and scalability”
[DailyPharm = Reporter Cha Ji-hyun] Heroes emerge in troubled times. Messenger ribonucleic acid (mRNA) vaccines are the biggest star born from the COVID-19 pandemic. Although the concept of mRNA technology emerged in the 1990s and stayed in the laboratory for a long time, it was realized for the first time during the pandemic. Since then, it has demonstrated rapid design and production and high flexibility, emerging as a next-generation platform that is changing the paradigm of biotherapeutics.
As global competition to secure leadership in mRNA technology intensifies, a domestic company has chosen to go head-to-head with its own technology. The four-year-old biotech SML Biopharm is that company. SML Biopharm aims to become a game-changer in the next-generation bio market by using mRNA platforms to treat diseases that were limited by existing therapies.
SML Biopharm has built two platforms: mRNA design technology that increases in vivo stability and protein expression efficiency, and AI-based lipid nanoparticle (LNP) optimization technology. Based on these, it is developing pipelines targeting various diseases including infectious diseases, cancer, and sarcopenia. Among these, an antibody therapeutic candidate for severe fever with thrombocytopenia syndrome (SFTS) is positioned as a key pipeline and development is being accelerated.
Founder and current CEO Nam Jae-hwan is an expert with more than 30 years of experience in vaccine research and biotech technology development. Nam served as a health researcher at the National Institute of Health, the predecessor of the Korea Disease Control and Prevention Agency under the Ministry of Health and Welfare, and currently serves as a professor in the Department of Biomedical Science at the Catholic University. I met Nam to hear about SML Biopharm’s core technologies and future vision.
What are the company’s main pipelines?
SML Biopharm is developing preventive vaccines and therapeutics based on mRNA technology. The main pipelines are being developed through two major approaches.
The first is using mRNA technology to induce immune responses to prevent or treat disease. Examples include mRNA cancer vaccines for cervical cancer and head and neck cancer caused by human papillomavirus (HPV), and a COVID-19 preventive vaccine. Both pipelines are at the preclinical stage. The COVID-19 preventive vaccine was selected in April of this year for the Korea Disease Control and Prevention Agency’s (KDCA) project to support mRNA vaccine development in preparation for pandemics and will receive support.
The second approach is to treat disease by directly expressing therapeutic proteins in the body via mRNA. A representative example is the antibody therapeutic candidate 'SBP301' being developed for SFTS. SBP301 delivers antibody genes in mRNA form to induce in vivo production of antiviral antibodies. Preclinical toxicology studies are currently underway.
Furthermore, the company has pipelines that express bispecific antibodies in vivo to treat cancer and a sarcopenia therapeutic pipeline that expresses a newly targeted muscle-regeneration-related protein. These pipelines have all completed proof of concept (POC) and are undergoing candidate optimization.
Please explain SFTS, the indication for the core pipeline.
SFTS is a viral infectious disease transmitted by the Haemaphysalis longicornis tick, known as the 'killer tick.' Cases continue to occur not only in Korea but also in East Asian regions such as China and Japan. Patients have continued to be reported this year in Korea, and recently a hospital in Cheongju experienced a cluster infection among medical staff due to human-to-human transmission.
Why did you start developing an SFTS therapeutic?
Although the number of SFTS patients is not large, it is a very severe disease with a fatality rate of around 20% when infected. Yet to date there are no approved treatments or vaccines. SML Biopharm decided to develop a therapeutic in light of the high fatality rate and the unmet medical need for SFTS. In particular, recognizing the necessity for early response from a public health perspective, the company sought to propose a new infectious disease response solution through an mRNA-based antibody therapeutic.
Introduce the SFTS therapeutic candidate SBP301.
SBP301 is a therapeutic that delivers the genetic information of an antibody with neutralizing activity against the SFTS virus in the form of mRNA into the body, inducing the body to produce the antibody itself. The antibody is based on antibodies derived from recovered patients.
SBP301 features high in vivo expression efficiency achieved using SML Biopharm’s proprietary mRNA platform. With support from the Ministry of Health and Welfare’s infectious disease prevention and treatment technology development program and the Ministry of Science and ICT’s national preclinical support system project, and with assistance from the Korea Research Institute of Bioscience and Biotechnology and the National Institute of Toxicological Research, preclinical toxicology studies are underway.
How does mRNA-based antibody expression differ from traditional antibody injection methods?
Conventional antibody therapies typically use mammalian cells to produce large quantities of antibody proteins outside the body, which are then administered by injection. This process takes a long time, is costly, and requires complex processes and stringent quality control.
In contrast, mRNA-based antibody therapeutics deliver the genetic information of the antibody in mRNA form, inducing cells in the human body to synthesize the antibody protein directly. In other words, the body’s cells are used as an 'antibody factory,' reducing separate protein production and purification processes and enabling more efficient and flexible production.
Additionally, mRNA can achieve sufficient effect with small amounts, and because design and production are fast, it has the advantage of rapid response to rapidly changing situations such as emerging infectious diseases or variant viruses.
What is the mid-to-long-term development strategy for SBP301?
SFTS continues to occur in Japan and China as well, and it is recognized in those regions as a disease with a high fatality rate. The company is considering expansion into Asian markets including those countries. Specifically, it has experience collaborating with a Japanese company regarding the introduction of antibody sequences. Through local validation and technology transfer, it aims to establish a foundation for global expansion.
There is also significant potential to expand to other indications. Since mRNA delivers the genetic information of proteins such as antibodies to be expressed directly in the body, it can express not only single antibodies but also bispecific antibodies and fusion proteins. There is room to expand indications to a variety of diseases beyond infectious diseases, including cancer, age-related diseases, and sarcopenia. While the company is currently focused on the SFTS indication, it is exploring pipeline expansion into anticancer therapies and sarcopenia therapeutics using the same platform.
Beyond simple therapeutic development, the expandability of the technology as a platform is noteworthy. What is the strategic value of the mRNA platform SML Biopharm is developing?
SML Biopharm’s mRNA platform is a versatile technology that can be extended in two directions: vaccines that induce immune responses (mRNA vaccines) and therapeutic protein expression (mRNA antibody therapeutics). It can be applied across various indications rather than being limited to a specific disease.
In particular, as experienced during the COVID-19 pandemic, mRNA offers rapid design and production and high flexibility, enabling quick responses in the early stages of infectious disease outbreaks. Beyond inducing immunity like a vaccine, it is also possible to directly express therapeutic proteins such as antibodies and bispecifics to use as treatments, making it a next-generation technology that can cover both prevention and treatment.
Because of these characteristics, when a novel infectious disease emerges, development can proceed rapidly once antibody or antigen sequences are secured, giving it great value as a national-level infectious disease response technology asset. SML Biopharm has formed a consortium with Biologics and Inventige Lab to participate in a national project aimed at securing domestic mRNA vaccine technology and strengthening vaccine sovereignty.
DailyPharm reporter Cha Ji-hyun (https://www.dailypharm.com/Users/News/NewsView.html?ID=325075)
