Rznomics Announces U.S. FDA Regenerative Medicine Advanced Therapy Designation Granted to 'RZ-001' for Hepatocellular Carcinoma

Rznomics, a South Korea-based biopharmaceutical company specializing in RNA-based gene therapeutics, announced on May 8, 2026, that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) Designation to RZ-001, its lead investigational drug for hepatocellular carcinoma (HCC). The RMAT designation was awarded based on promising Phase 1b/2a clinical trial data, including RZ-001’s safety profile and preliminary response rates, which were presented at the American Association for Cancer Research (AACR) Annual Meeting in April 2026. RZ-001 operates through Rznomics’ proprietary trans-splicing ribozyme technology platform, which replaces cancer-specific RNA with therapeutic RNA. This dual-action approach enhances tumor selectivity and safety, offering a novel mechanism for treating HCC, a condition with limited treatment options. The drug previously received Orphan Drug Designation (ODD) in 2024 and Fast Track Designation (FTD) in 2025 for HCC. The FDA’s RMAT designation underscores the clinical potential of RZ-001 and will allow Rznomics to accelerate development efforts in the U.S. The company plans to engage in formal discussions with the FDA regarding clinical trial design, Chemistry, Manufacturing, and Controls (CMC), and commercialization strategies. Sung-woo Hong, Vice President of Rznomics, stated that the designation will help expedite partnerships and development initiatives. Seong-Wook Lee, CEO of Rznomics, emphasized that the RMAT designation validates the innovation of the company’s RNA editing platform. He noted that the company will focus on global development and commercialization to deliver a breakthrough therapeutic for HCC, a disease with significant unmet medical needs. Introduced under the 21st Century Cures Act in 2016, RMAT designation supports the development of regenerative medicine therapies, including cell and gene therapies, for serious conditions with unmet medical needs. Rznomics, which listed on the KOSDAQ market in December 2025, also collaborates with Eli Lilly under a research agreement signed in May 2025 for RNA editing therapeutics.