Rznomics, a clinical-stage biopharmaceutical company focused on RNA-based gene therapeutics, has announced that the US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead investigational candidate, RZ-001, for the treatment of Hepatocellular Carcinoma (HCC).

The RMAT designation is a specialised FDA programme aimed at accelerating the development and review of innovative regenerative medicine therapies intended to treat serious or life-threatening diseases. The designation is granted to therapies supported by preliminary clinical evidence indicating their potential to address unmet medical needs. The programme also enables enhanced FDA guidance, rolling reviews, priority review eligibility and accelerated approval pathways, helping to speed up patient access to advanced therapies.

RZ-001 is a next-generation oncology therapeutic developed using Rznomics’ proprietary trans-splicing ribozyme technology platform. The therapy works by replacing cancer-specific RNA with therapeutic RNA, offering a novel mechanism designed to overcome limitations associated with conventional cancer treatments. According to the company, the dual-action approach improves tumour selectivity while enhancing safety, positioning the therapy as a potential treatment option for HCC patients with limited alternatives.

The candidate had earlier received Orphan Drug Designation in 2024 and Fast Track Designation in 2025 from the FDA for the treatment of hepatocellular carcinoma. The latest RMAT designation follows encouraging interim clinical data from an ongoing phase 1b/2a trial that was presented at the American Association for Cancer Research (AACR) Annual Meeting in April 2026.

Commenting on the development, Sung-woo Hong, Vice President of Rznomics, said the RMAT designation would support the company’s plans to accelerate its US development and partnership initiatives, including discussions with the FDA on clinical trial design, Chemistry, Manufacturing and Controls (CMC) and commercialisation strategies.

Seong-Wook Lee, Chief Executive Officer of Rznomics, said the FDA’s recognition validates the innovation and competitiveness of the company’s RNA editing platform. He added that the company plans to intensify efforts toward global development and commercialisation of RZ-001 to address the significant unmet medical needs in liver cancer treatment.

Introduced under the 21st Century Cures Act in 2016, the RMAT designation programme was created to encourage the development of regenerative medicine products, including gene and cell therapies, tissue-engineered products and combination therapies. Therapies eligible for the programme must demonstrate the potential to treat serious conditions with limited treatment options.

Founded in South Korea, Rznomics specialises in RNA-based gene therapies with applications across multiple disease areas. The company entered into a research collaboration and licensing agreement with Eli Lilly in May 2025 for the development of a novel RNA editing therapeutic and was listed on the KOSDAQ market in December 2025.