Rznomics’ RZ-001 RNA-editing therapeutic earns FDA RMAT designation for hepatocellular carcinoma

Edited by Robert McTiernan··3 min read

Rznomics (KOSDAQ: 476830), a clinical-stage biopharmaceutical company based in South Korea, announced that the US FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RZ-001, its lead investigational RNA-editing gene therapeutic, for the treatment of hepatocellular carcinoma (HCC). The designation follows interim Phase Ib/IIa data presented at the American Association for Cancer Research (AACR) Annual Meeting in April 2026.

RMAT designation, introduced under the 21st Century Cures Act in 2016, applies to regenerative medicine therapies — including cell and gene therapies — intended to treat serious or life-threatening conditions, where preliminary clinical evidence indicates potential to address unmet medical needs. It confers eligibility for rolling and priority review, as well as more frequent FDA interactions on trial design, manufacturing, and commercialization strategy. RZ-001 had previously received Orphan Drug Designation (ODD) in 2024 and Fast Track Designation (FTD) in 2025, both for HCC.

RZ-001 is built on Rznomics’ proprietary trans-splicing ribozyme platform, which edits RNA at the transcript level by replacing cancer-specific RNA with therapeutic RNA. The mechanism is distinct from conventional small-molecule or antibody-based approaches in that it operates upstream of protein expression, targeting the RNA transcript rather than the encoded protein or a cell-surface receptor. The company describes the platform as having dual-action properties — tumor selectivity through recognition of cancer-specific transcripts, and a therapeutic payload delivered via the replaced RNA sequence.

The clinical data underpinning the RMAT designation were drawn from an open-label, multicenter Phase Ib/IIa study evaluating RZ-001 in combination with atezolizumab and bevacizumab in patients with human telomerase reverse transcriptase (hTERT)-positive HCC, conducted across nine institutions in South Korea. At the AACR Annual Meeting in April 2026, Rznomics reported a confirmed objective response rate (ORR) of 38.5% by RECIST v1.1 criteria, rising to 46.2% when unconfirmed responses were included. Using modified RECIST (mRECIST), which accounts for tumor necrosis and viability and is considered more informative in HCC, the ORR reached 61.5%, including at least one complete response. The company characterized the safety profile as favorable.

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The combination design is relevant to interpreting these figures. Atezolizumab, a PD-L1 inhibitor developed by Roche, and bevacizumab, a VEGF-targeting antibody, together constitute the Tecentriq (atezolizumab)/Avastin (bevacizumab) regimen that received US FDA approval in 2020 as a first-line treatment for unresectable HCC, based on the IMbrave150 trial which reported an ORR of approximately 27% by RECIST v1.1. The 38.5% confirmed ORR observed with RZ-001 added to this backbone is directionally higher, though cross-trial comparisons are limited by differences in patient selection, prior treatment history, and study design. The Phase Ib/IIa study was not powered for efficacy, and the patient numbers were not disclosed in available public materials.

The hTERT-positive patient selection criterion used in the RZ-001 studies is also notable. hTERT is expressed in the majority of HCC tumors but not in normal hepatic tissue, which provides a basis for tumor-selective RNA targeting. This biomarker-defined approach contrasts with the broader patient selection used in most approved HCC regimens, and its implications for trial design and eventual labeling will likely be a focus of FDA discussions enabled by the RMAT designation.

Rznomics listed on the KOSDAQ market in December 2025 and signed a research collaboration and license agreement with Eli Lilly in May 2025 covering a separate RNA editing therapeutic program. The company has indicated it intends to use the RMAT designation to initiate formal FDA discussions on clinical trial design, chemistry, manufacturing, and controls, and commercialization strategy for RZ-001. An active combination study, NCT06695026, evaluating RZ-001 with valganciclovir and atezolizumab/bevacizumab in hTERT-positive HCC, is currently recruiting.

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