Safer Than Direct Gene Therapy, Enhanced Specificity for Anticancer Drug Development

Aiming for IPO Through Licensing-Out Within the Year, "Platform Deals Also Possible"

"We aim to develop the world's first treatment for incurable cancers using RNA editing technology."

In an interview on March 7, Seongwook Lee, CEO of Rznomics, stated, "Our proprietary RNA editing platform, Trans-Splicing Ribozyme (TSR), simultaneously expresses therapeutic RNA while suppressing unintended RNA expression."

Rznomics was founded in 2017 after CEO Lee dedicated over 20 years to TSR research. He began studying TSR in the 1990s as a postdoctoral researcher at Duke Medical Center in the U.S., where the first research papers on TSR were published.

However, the emergence of RNA interference (RNAi) in the 2000s led to a decline in TSR development momentum. As global interest in RNAi-based therapeutics surged, many scientists who had been working on TSR shifted their focus to RNAi research. Lee returned to Korea and continued his TSR research as a professor at Dankook University.

"When TSR was developed at first, it lacked specificity and efficacy, and there was little research on delivery vectors for in vivo applications," he explained. "However, in the mid-2000s, through advancements in bioengineering, we achieved the ability to target specific genes for cancer treatment. The emergence of gene therapies utilizing viral vectors as drug delivery systems further accelerated TSR development."

A Safer Alternative to Gene Editing

Gene-editing therapies such as CRISPR gene scissors pose concerns about irreversible genetic modifications if unintended targets are altered. Furthermore, there are currently no treatments that enable gene editing inside the human body post-administration. RNAi therapies also face limitations, as existing drug delivery systems are primarily designed for targeting liver cells, requiring further research for development as anticancer drugs.

On the other hand, TSR does not directly modify DNA. Instead, it removes targeted RNA and replaces it with therapeutic RNA. Rznomics designs optimized drug delivery systems for each disease, allowing precise targeting of the intended site. Depending on the indication, adenoviral vectors are used for temporary effects, while adeno-associated virus (AAV) vectors are used for long-lasting, one-time treatments.

Lead Pipeline: RZ-001

Rznomics’ lead pipeline candidate is RZ-001, an anticancer drug currently in clinical trials. In both the U.S. and Korea, RZ-001 is undergoing Phase 1b/2a trials for hepatocellular carcinoma (HCC) and Phase 1/2a trials for glioblastoma (GBM). The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for both indications. Additionally, the glioblastoma program has been designated under the Expanded Access Program (EAP), which allows patients with no available treatment options to receive the investigational drug.

RZ-001 is also set to enter combination trials with immune checkpoint inhibitors. Rznomics has secured free-of-charge supply agreements for Roche’s Tecentriq and Celltrion’s Vegzelma (Avastin biosimilar) for domestic combination studies with RZ-001. Tecentriq and Avastin have already been approved by the FDA as a first-line treatment for hepatocellular carcinoma.

"In preclinical studies, RZ-001 showed improvements in biomarkers associated with immune checkpoint inhibitor responses," Lee said. "Due to the anticipated synergy with Tecentriq and Avastin, we were able to secure these drugs free of charge for our trials."

IPO Target and Global Licensing Plans

Rznomics aims to list on the KOSDAQ stock market through a special listing track as early as this year or by next year at the latest. "Our TSR technology offers not only pipeline assets but also platform licensing opportunities," Lee emphasized. "We plan to achieve licensing-out deals within the year and apply for a technology evaluation to proceed with our IPO."