"Without deep science, supported by rational evidence and global competitiveness, it is challenging to win the hearts of investors."

Rznomics CEO Seongwook Lee is pioneering the field of RNA-based therapeutics through scientific rigor. In a recent interview with Hit News, he elaborated on the strengths of the company’s Trans-splicing Ribozyme technology, its circular RNA platform, and a vision for expanding therapeutic indications based on global competitiveness. Last month, Rznomics successfully secured KRW 20.3 billion in Pre-IPO funding.

Rznomics is gaining attention in the field of RNA-based therapeutics for its innovative technology. The company’s core Trans-splicing Ribozyme platform replaces mutated RNA with normal RNA, enabling the production of functional proteins. Unlike conventional DNA-based gene therapies, which alter entire genomes, this technology selectively edits or replaces only the specific RNA implicated in disease.

"Our technology is reversible, making it safer and more controllable compared to DNA therapies, which carry the risk of permanently altering cellular genes," said CEO Lee.

By focusing on targeted RNA segments instead of delivering entire RNA genomes, Rznomics optimizes the use of Adeno-associated virus (AAV) vectors. "Although AAV vectors have size limitations for delivered genomes, our technology’s selective RNA replacement approach minimizes these constraints," Lee explained. He added that AAV vectors demonstrate stable activity in non-dividing cells and enable prolonged RNA expression, particularly benefiting diseases such as central nervous system (CNS) and retinal disorders, where long-term treatments are crucial.

Describing the therapeutic mechanism, Lee likened it to installing a factory within non-dividing cells: "By administering AAV, we can continuously produce therapeutic RNA (Trans-splicing Ribozyme). A single administration could provide sustained therapeutic effects."

For diseases like cancer, where genetic material doesn’t need to remain long-term, Lee emphasized the advantages of RNA-based therapies, which achieve therapeutic effects while minimizing side effects. For such indications, Rznomics uses adenovirus vectors, which work temporarily without integrating into the chromosome—making them ideal for cancer treatment.

Another strength of Rznomics' technology is its ability to address diverse mutations with a single drug. "Even if patients have mutations in different regions, we can map specific RNA sequences and develop a single therapy to correct multiple mutations," Lee said.

The platform also avoids risks associated with overexpression by controlling RNA expression levels. Traditional RNA therapies often trigger off-target effects or overexpression, leading to undesirable side effects. In contrast, "Our technology ensures target specificity by replacing only the intended RNA. This allows the production of proteins at normal levels, effectively eliminating the risk of overexpression," Lee explained.

Highlighting the platform’s versatility, Lee stated, "Rznomics' technology is modular. If a target RNA is identified, a corresponding therapeutic RNA can be developed, paving the way for new treatments in incurable and rare diseases with unmet medical needs."

In addition to Trans-splicing Ribozyme technology, Rznomics is developing a cutting-edge circular RNA platform. Unlike linear RNA, circular RNA forms a closed-loop structure with no open ends, making it resistant to degradation and enabling long-lasting functionality within cells.

"When linear RNA is used as a therapeutic, modified nucleotides are often needed to suppress immune responses. However, circular RNA works safely in cells without modifications," Lee explained.

Rznomics’ proprietary circular RNA technology eliminates unnecessary sequences and is composed entirely of the desired gene of interest (GOI), enhancing cellular safety. Lee noted that circular RNA has potential applications in advanced cell and gene therapies, including in vivo CAR-T therapies, simplifying the manufacturing process by bypassing complex cell manipulation steps.

However, Lee acknowledged that circular RNA remains in its early stages and requires further research and development to prove its commercial viability. "Leading groups in the U.S. are exploring applications of circular RNA in areas like vaccines and in vivo CAR-T therapies. However, identifying the most competitive use case for this technology and addressing challenges in large-scale production remain key tasks," he said.

Rznomics' innovations in RNA-based therapies have earned recognition from the global scientific and pharmaceutical communities. The company’s groundbreaking research has been featured in leading journals such as Nature Biotechnology and Nature, underscoring its scientific credibility.

"The trust we’ve gained from the global academic and industrial communities is a significant asset," Lee said, emphasizing the role of scientific validation in attracting investments and driving the company’s growth.

Rznomics has also secured Orphan Drug Designation (ODD) and Fast Track Designation from the U.S. Food and Drug Administration (FDA), further solidifying its commercial potential. "Recognition from the FDA sends a strong signal to global pharmaceutical companies and supports our independent market entry efforts," Lee noted.

Building on these achievements, Rznomics is actively collaborating with global pharmaceutical companies to accelerate commercialization. "We are establishing partnerships with several companies to expedite market entry while simultaneously validating and commercializing our technologies," Lee said.

Rznomics remains committed to applying its innovations to improve patient outcomes. "Our goal is to provide better treatment options for patients by expanding the potential of our platform," Lee concluded, reaffirming the company’s vision to lead the global RNA therapeutics market with its differentiated competitive edge.