Rznomics, Inc.

RMAT designation based on promising Phase 1b/2a clinical data, including safety profile and preliminary response rates, with trans-splicing ribozyme-based RNA editing platform SEOUL, South Korea, May 8, 2026 /PRNewswire/ -- Rznomics a biopharmaceutical company specializing in RNA-based gene therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) Designation to RZ-001, its lead investigational candidate for the treatment of hepatocellular carcinoma (HCC). RMAT designation is a specialized FDA program created to accelerate the development and review of promising new therapies, including gene therapies, intended to treat serious or life-threatening conditions. Applicant is required to submit preliminary clinical evidence suggesting the potential to address unmet medical needs. This designation provides important opportunities during the drug development process, including increased FDA guidance and eligibility for priority and rolling reviews, as well as accelerated approval pathways. By streamlining these regulatory milestones, the program aims to bring transformative innovations to patients more quickly. RZ-001 is the next-generation oncology therapeutics based on Rznomics' proprietary trans-splicing ribozyme technology platform. By replacing cancer-specific RNA with therapeutic RNA, RZ-001 offers a novel mechanism of action designed to overcome the limitations of conventional therapies. The platform's dual-action approach—enhancing both tumor selectivity and safety—presents a promising new option for HCC patients with limited treatment alternatives. RZ-001 previously received Orphan Drug Designation (ODD) in 2024 and Fast Track Designation (FTD) in 2025 for the treatment of HCC. The FDA's decision to grant RMAT status highlights the clinical potential and innovativeness of RZ-001, particularly following the meaningful interim signals from the Phase 1b/2a clinical trial presented at the American Association for Cancer Research (AACR) Annual Meeting in April 2026. "With the RMAT designation, we plan to accelerate our U.S. development and partnership initiatives by initiating formal discussions with the FDA regarding clinical trial design, Chemistry, Manufacturing, and Controls (CMC), and commercialization strategies," said Sung-woo Hong, Vice President of Rznomics. Seong-Wook Lee, CEO of Rznomics, added, "Receiving RMAT designation for RZ-001 is a profound validation of the innovation and competitiveness of our RNA editing platform by the FDA. We will concentrate our resources on global development and commercialization to provide a breakthrough therapeutic option in the field of HCC, where unmet medical needs remain exceptionally high." About RMAT Designation Introduced under the 21st Century Cures Act in 2016, the RMAT designation was established to foster the development of innovative regenerative medicine therapies and expand patient access. The program encompasses cell and gene therapies, therapeutic tissue engineering products, and combination products. To be eligible, a drug must be a regenerative medicine therapy intended to treat serious conditions, with preliminary clinical evidence indicating that the drug has the potential to address unmet medical needs for such a condition. About Rznomics Inc. Rznomics is a clinical-stage biopharmaceutical company based in South Korea focused on developing RNA-based gene therapies. The company's proprietary trans-splicing ribozyme platform enables precise RNA editing and has broad applicability across multiple indications. The company signed a research collaboration and license agreement with global pharmaceutical company Eli Lilly in May 2025 for the development of a novel RNA editing therapeutic and listed on the KOSDAQ market in December 2025. (KOSDAQ 476830) For more information, please visit SOURCE Rznomics 21% more press release views with Request a Demo

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. 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. This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: https://allsci.com/news/ Your email address will not be published. Required fields are marked * Comment * Name * Email * Website Privacy Terms About Us Copyright © 2026. AllSci Corp. All rights reserved.

SEOUL, South Korea, May 4, 2026 – Rznomics Inc. (CEO Seong-Wook Lee, KOSDAQ 476830), a biopharmaceutical company specializing in RNA-based gene therapeutics, announced today its participation in the 29th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, to be held in Boston from May 11 to 15. SEOUL, South Korea, May 4, 2026 – Rznomics Inc. (CEO Seong-Wook Lee, KOSDAQ 476830), a biopharmaceutical company specializing in RNA-based gene therapeutics, announced today its participation in the 29th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, to be held in Boston from May 11 to 15. Rznomics will present a total of three abstracts, including one oral presentation and two poster presentations. The company aims to showcase its latest research milestones in proprietary circular RNA (circRNA) technology and its novel oncology gene therapy platforms. Rznomics will present a total of three abstracts, including one oral presentation and two poster presentations. The company aims to showcase its latest research milestones in proprietary circular RNA (circRNA) technology and its novel oncology gene therapy platforms. ASGCT is the world’s largest professional organization for gene and cell therapy. Its annual meeting serves as a premier global stage for sharing innovative technologies and clinical outcomes. The event continues to grow in scale, with over 8,000 registrants and 2,000 abstracts submitted in 2025. ASGCT is the world’s largest professional organization for gene and cell therapy. Its annual meeting serves as a premier global stage for sharing innovative technologies and clinical outcomes. The event continues to grow in scale, with over 8,000 registrants and 2,000 abstracts submitted in 2025. In the oral presentation, Rznomics will introduce its research on polymeric nanocarriers designed for the selective systemic delivery of circular RNA to splenic immune cells. Rznomics’ circRNA is characterized by its high stability, sustained expression, and the absence of residual exogenous sequences. Notably, this technology is gaining significant attention in the industry as a potential in vivo CAR-T platform, capable of generating CAR-T cells directly within the body. In the oral presentation, Rznomics will introduce its research on polymeric nanocarriers designed for the selective systemic delivery of circular RNA to splenic immune cells. Rznomics’ circRNA is characterized by its high stability, sustained expression, and the absence of residual exogenous sequences. Notably, this technology is gaining significant attention in the industry as a potential in vivo CAR-T platform, capable of generating CAR-T cells directly within the body. The poster presentations will highlight: The poster presentations will highlight: RNA Structure Engineering: A study on self-targeting and splicing (STS) reactions that enable the efficient circularization of long-form RNAs, which are traditionally difficult to circularize. RNA Structure Engineering: A study on self-targeting and splicing (STS) reactions that enable the efficient circularization of long-form RNAs, which are traditionally difficult to circularize. Next-Gen Oncology Platform: A novel strategy using RNA trans-splicing ribozyme technology. This platform achieves a synergistic therapeutic effect by concurrently expressing therapeutic genes and immune checkpoint inhibitors (ICIs) specifically within cancer cells. Next-Gen Oncology Platform: A novel strategy using RNA trans-splicing ribozyme technology. This platform achieves a synergistic therapeutic effect by concurrently expressing therapeutic genes and immune checkpoint inhibitors (ICIs) specifically within cancer cells. “Presenting our latest research at ASGCT, where world-class researchers in gene and cell therapy gather, is a significant milestone,” said Seong-Wook Lee, CEO of Rznomics. “We look forward to demonstrating the differentiation and scalability of our circRNA and trans-splicing ribozyme platform on the international stage, further solidifying our competitive edge in the next-generation RNA therapeutics market.” “Presenting our latest research at ASGCT, where world-class researchers in gene and cell therapy gather, is a significant milestone,” said Seong-Wook Lee, CEO of Rznomics. “We look forward to demonstrating the differentiation and scalability of our circRNA and trans-splicing ribozyme platform on the international stage, further solidifying our competitive edge in the next-generation RNA therapeutics market.” In accordance with ASGCT embargo policies, oral presentation materials will be released at 6:00 AM ET on the day of the presentation, and posters will be available at 6:00 AM ET on May 12, 2026. In accordance with ASGCT embargo policies, oral presentation materials will be released at 6:00 AM ET on the day of the presentation, and posters will be available at 6:00 AM ET on May 12, 2026. 1. Oral Presentation 1. Oral Presentation Title: Intravenous Delivery of Circular RNAs to Splenic Immune Cells Using Polyaspartamide-Based Polyplexes Title: Intravenous Delivery of Circular RNAs to Splenic Immune Cells Using Polyaspartamide-Based Polyplexes Abstract Number: 336 Abstract Number: 336 Date & Time: May 14, 2026; 11:15 – 11:30 AM ET Date & Time: May 14, 2026; 11:15 – 11:30 AM ET Location: MCEC Room 206AB (Level 2) Location: MCEC Room 206AB (Level 2) 2. Poster Presentation 2. Poster Presentation Title: Engineering of P1 Construct for Efficient Circular RNA Generation by End-to-End Self-Targeting and Splicing (STS) Reaction for Long circRNAs Title: Engineering of P1 Construct for Efficient Circular RNA Generation by End-to-End Self-Targeting and Splicing (STS) Reaction for Long circRNAs Abstract Number: 2122 Abstract Number: 2122 Date & Time: May 13, 2026; 5:00 – 6:30 PM ET Date & Time: May 13, 2026; 5:00 – 6:30 PM ET Location: Poster Hall Location: Poster Hall 3. Poster Presentation 3. Poster Presentation Title: TERT mRNA Reprogramming for Dual Cancer Therapy: Concurrent Expression of HSVtk and Immune Checkpoint Inhibitor via Trans-Splicing Ribozyme Title: TERT mRNA Reprogramming for Dual Cancer Therapy: Concurrent Expression of HSVtk and Immune Checkpoint Inhibitor via Trans-Splicing Ribozyme Abstract Number: 3317 Abstract Number: 3317 Date & Time: May 14, 2026; 5:00 – 6:30 PM ET Date & Time: May 14, 2026; 5:00 – 6:30 PM ET Location: Poster Hall Location: Poster Hall