Rznomics said on the 8th that RZ-001, which is being developed as a treatment for primary hepatocellular carcinoma (HCC), has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA).
RMAT is an expedited development program granted to candidates among regenerative therapies for serious diseases that have demonstrated innovation and therapeutic potential in early clinical trials. Designation allows benefits such as priority review, rolling review, and accelerated approval.
RZ-001 is an anticancer gene therapy that applies Rznomics' ribozyme-based RNA editing platform technology, which selectively cleaves specific RNA. It is designed to selectively target only cancer cells, aiming to improve safety and efficacy compared with existing therapies. The therapy previously received orphan drug designation and fast track designation from the FDA.
The company said that interim results from phase 1b/2a, unveiled at the American Association for Cancer Research (AACR 2026) in April, positively influenced the RMAT designation.
Rznomics plans to use this designation as a springboard to begin full-fledged discussions with the FDA on clinical design, chemistry, manufacturing and controls (CMC), and commercialization strategy.
Lee Sung-wook, CEO of Rznomics, said, "This RMAT designation recognizes the innovation and competitiveness of our RNA editing platform by the FDA," adding, "We will focus our capabilities on global development and commercialization to present an innovative treatment option in liver cancer, where there is significant unmet medical need."