[Bio Story] ①Algenomics Takes Aim at New Drug Development with RNA Editing Platform

Interview with Lee Sungwook, CEO of Algenomics ①

Commercializing an RNA Editing Platform After 20 Years of Research
Suppressing Mutant RNA and Expressing Therapeutic Normal RNA
Simultaneous Regulation Through a Single Agent

"Gene-editing scissors directly intervene in the DNA, which is the cell's original blueprint. This means a single misstep could result in permanent side effects. In contrast, the technology developed by Algenomics takes a much safer approach by preserving the original blueprint and editing its copy—RNA—instead."

Seonguk Lee, CEO of Algenomics, summarized the key competitive advantage of the company’s ribonucleic acid (RNA) substitution enzyme-based platform in this way during an interview with The Asia Business Daily at the company's headquarters in Pangyo, Seongnam, Gyeonggi Province, on July 22, 2026. Algenomics is pioneering new pathways for the treatment of intractable diseases with its proprietary RNA editing technology, which offers solutions to the limitations of conventional gene therapy markets. Globally, Algenomics is the only company that has successfully commercialized and platformized this technology.

The greatest feature of this platform is its ability to both identify and excise disease-causing mutant RNA, while also seamlessly attaching therapeutic RNA in its place—all with a single molecule. This is in stark contrast to existing technologies: small interfering RNA (siRNA) only suppresses the expression of disease genes, and messenger RNA (mRNA) simply increases gene expression. The platform is especially effective in conditions like dominant hereditary diseases, which require both suppression of mutant gene function and supplementation with normal genes.

Lee explained, "Some diseases can't be treated just by suppressing gene expression, so it is normally necessary to use both inhibitors and enhancers. But with our therapy, we achieve both goals with a single treatment." He continued, "Most importantly, the technology precisely regulates only the necessary amount at the exact location of the target gene, fundamentally blocking toxicity or side effects caused by gene overexpression."

Algenomics has also overcome a long-standing limitation—because RNA molecules themselves exist only temporarily in the body and then disappear, repeated treatments were previously essential. The company solved this with a proprietary delivery system: instead of injecting therapeutic RNA directly into a patient, they deliver DNA that continuously produces therapeutic RNA, using viral vectors to transfer it into cells. This effectively establishes a ‘factory’ inside the body, allowing the therapeutic agent to be produced continuously with just a single administration.

Lee emphasized, "The biggest drawback of RNA is that it is quickly synthesized and then disappears, requiring constant administration. But we use a DNA-based virus that continuously produces the gene therapy agent. The delivered DNA remains permanently within the target cells, prolonging the treatment’s effect."

Diversifying the delivery system according to therapeutic objectives and disease characteristics is also a major strength of the platform. For anticancer agents that need to act powerfully in a short time and then disappear from the body, adenoviruses are used. For chronic indications requiring sustained efficacy throughout a patient’s lifetime—such as central nervous system or hereditary retinal disorders—adeno-associated virus (AAV) vectors are adopted, allowing for precise, tailored design.

It took nearly 20 years of patience and optimization to establish the current platform. Lee first discovered the potential of this technology in 1995 during his postdoctoral work in the United States. While the original idea emerged at that time, it initially lacked sufficient efficacy and safety for therapeutic use, and the technical infrastructure to deliver agents into the body was absent. Even within the industry, interest and funding flowed toward siRNA, making further research in the U.S. difficult. Nevertheless, Lee brought the technology to Korea, and after two decades of persistent optimization, he founded Algenomics in 2017.

Lee stated, "This was a niche field where no one dared attempt commercialization. But by refusing to give up, I refined the platform to operate both safely and effectively on patient cells, making it our unique asset today." He continued, "As delivery technologies for gene therapies advanced and the U.S. Food and Drug Administration (FDA) began granting approvals in the late 2010s, the environment finally emerged for the technology we had accumulated over many years to shine." Lee added, "Our platform can be expanded to correct any genetic target at the RNA level. Going forward, it is an infinitely scalable and versatile platform that will prove its competitive edge on the global stage for indications ranging from cancers to central nervous system diseases."

This content was produced with the assistance of AI translation services.