Rznomics

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Rznomics Discloses Interim Clinical Abstract for RNA Oncology Drug ‘RZ-001’ in Hepatocellular Carcinoma at AACR 2026

Rznomics Inc., a biotechnology company specializing in RNA-based gene therapeutics, announced that it will deliver an oral presentation on the interim Phase 1/2a clinical results for ‘RZ-001’—an RNA-based oncology candidate targeting hepatocellular carcinoma (HCC)—at the AACR Annual Meeting 2026, commencing on April 17 (local time). Following the expiration of the official embargo, the clinical abstract has been released.The presentation is based on data from an ongoing clinical trial involving systemic treatment-naive HCC patients who are refractory to or ineligible for Transarterial Chemoembolization (TACE).According to the disclosed abstract, the combination therapy of RZ-001 with atezolizumab and bevacizumab achieved an Objective Response Rate (ORR) of 38.5% based on RECIST v1.1 and 53.8% based on mRECIST. Notably, the Complete Response (CR) rate reached 23% (mRECIST). These figures represent a significant improvement over the IMbrave150 study—the pivotal trial for the current first-line standard of care (atezolizumab/bevacizumab)—showing an ORR increase of over 10% and a CR rate more than double that of the previous landmark study.In HCC clinical evaluations, mRECIST (modified RECIST) is a critical metric alongside RECIST, as it assesses "viable tumor" activity. Since tumors may exhibit internal necrosis (loss of activity) despite limited size reduction, mRECIST provides a more precise evaluation of therapeutic efficacy."Due to the interval between the abstract submission and the conference, we will present even more updated clinical data during our oral session on April 19," said Sung-woo Hong, Vice President of Rznomics. "While these interim results are highly encouraging, we remain cautious and advise against direct comparison with other clinical trials or overinterpretation at this stage."RZ-001 has been granted Orphan Drug Designation (ODD) by the U.S. FDA. This status provides strategic advantages for global commercialization, including clinical trial tax credits, waivers of FDA user fees, and seven years of market exclusivity upon approval.

2026-04-18
Rznomics and Inha University Announce Research Breakthrough in Circular RNA Delivery for ‘In Vivo CAR-T’ Applications

Rznomics Inc, a RNA-based gene therapeutics leading company, announced that its joint research with Professor Hyun Jin Kim’s team at Inha University has been published in a prestigious international journal. The study focuses on a novel RNA delivery strategy capable of directly targeting immune cells within the body (in vivo). The research, titled “Delivery of Circular RNAs into Splenic Immune Cells via Intravenous Administration of Polyaspartamide Derivative Polyplexes,” was published online on March 27, 2026, in ACS Biomaterials Science & Engineering, a global journal published by the American Chemical Society.This study evaluated the potential of delivering circular RNA (circRNA) to the spleen—an organ rich in immune cells—through simple intravenous (IV) administration. The approach combines Rznomics’ proprietary circular RNA technology with Inha University’s polyamino acid-based polymer carriers.While conventional CAR-T therapies have shown revolutionary success in treating hematologic malignancies by genetically re-engineering a patient’s immune cells ex vivo, they are hindered by complex manufacturing processes, arduous administration protocols, and high costs. Consequently, the ‘in vivo CAR-T’ approach—which directly delivers CAR genes to immune cells inside the patient—is emerging as a next-generation strategy for oncology and autoimmune diseases due to its potential to simplify manufacturing, reduce costs, and significantly improve patient access.Rznomics’ circular RNA features a "clean structure" devoid of unnecessary exogenous sequences, providing superior stability and sustained expression. When paired with Professor Kim’s cationic polymer carrier, which enhances endosomal escape and particle stability, the intracellular delivery efficiency of the RNA is maximized.The research results demonstrated that the Rznomics circular RNA-polymer complex was delivered to various immune cells with exceptional efficiency, showing high selectivity for the spleen upon IV administration. Notably, delivery to the spleen was 10 times higher than to the liver. Furthermore, the circular RNA exhibited twice the half-life and three times the T-cell delivery efficiency in splenic tissue compared to conventional linear RNA. These findings suggest a viable path for creating next-generation CAR-T cell therapies without the need for external cell manipulation."Based on this technology, we will continue to explore the potential applications of polyamino acid polymer-based gene therapies through follow-up studies," said Professor Hyun Jin Kim.Dr. Seong-Wook Lee, CEO of Rznomics commented, "This industry-academic collaboration confirms that our circular RNA technology can be effectively applied to target-specific gene therapies for immune cells in vivo. We plan to continuously expand the scope of our applications to include CAR-T and other advanced therapeutic modalities."This research was supported by the Bio & Medical Technology Development Program under the National Research Foundation of Korea (NRF) and the Basic Research Laboratory Support Program.

2026-03-30
Rznomics to Present Oral Abstract of Clinical Data for its Anticancer Gene Therapy at AACR 2026

Rznomics (CEO Seong-Wook Lee, KOSDAQ 476830), a biotechnology company specializing in the development of RNA-based gene therapies, has announced the title of its oral presentation abstract for the American Association for Cancer Research (AACR) 2026, held in San Diego from April 17 to 22.The presentation is titled: "A phase 1b/2a open-label, multicenter, randomized, dose escalation study evaluating the safety, tolerability and efficacy of RZ-001 in combination with valganciclovir (VGCV) and atezolizumab/bevacizumab in subjects with hepatocellular carcinoma."The presentation, scheduled for April 19, will be delivered by Professor Yoon Jun Kim from the Department of Gastroenterology at Seoul National University Hospital. It will unveil interim safety and efficacy results from the ongoing clinical trial of RZ-001 for the treatment of hepatocellular carcinoma (HCC).A representative from Rznomics stated, "While the interim results of RZ-001 are a major highlight of this AACR presentation, the more significant implication lies in the clinical validation of our platform. This serves as a critical testbed to secure Clinical Proof of Concept (PoC) for Rznomics' proprietary Trans-splicing Ribozyme technology, demonstrating its potential beyond a single pipeline."The flagship pipeline, RZ-001, is a multi-mechanistic anticancer gene therapy that targets telomerase mRNA, which is expressed in over 80% of all cancer types. It is designed to induce cancer cell apoptosis, promote immune cell infiltration, and enhance response rates to immunotherapies. RZ-001 has received Investigational New Drug (IND) approval from the South Korean Ministry of Food and Drug Safety (MFDS) and the U.S. Food and Drug Administration (FDA). Furthermore, it has been granted 'Fast Track' and 'Orphan Drug Designation (ODD)' by the U.S. FDA for both primary liver cancer (HCC) and glioblastoma (GBM).Currently, Rznomics is conducting a Phase 1b/2a clinical trial evaluating RZ-001 in combination with immunotherapy for HCC patients, with patient recruitment and dosing underway across nine institutions in South Korea. The combination agents, Atezolizumab and Bevacizumab, are the primary first-line treatments used in over 70% of advanced HCC cases. However, given that the standard of care (SoC) response rate remains at approximately 20-30%. Rznomics expects significant commercial synergy if the data supports improved clinical efficacy and response rates through the RZ-001 combination.Moreover, since RZ-001 targets telomerase mRNA—prevalent in the majority of malignancies—it holds immense potential for indication expansion across a wide array of solid tumors.

2026-03-18
Rznomics to Lead Next-Generation Gene Therapy with Trans-splicing Ribozyme Platform

Rznomics Inc. (CEO Seong-Wook Lee) held an Investor Relations (IR) conference on the 3rd December, attended by the CEO and key executives, to share its strategic roadmap and vision following its upcoming listing on the KOSDAQ market.Founded in 2017, Rznomics specializes in developing innovative therapies for cancer and rare intractable diseases using its proprietary RNA replacement enzyme-based RNA editing and correction platform. This platform is uniquely capable of performing three functions—targeting RNA cleavage, splicing, and replacement—within a single therapeutic agent. The technology is garnering significant attention from global pharmaceutical companies and academia due to its scalability across various mutations, its safety, and its optimized delivery efficiency tailored to specific indications.The company’s lead pipeline asset, RZ-001, targets hepatocellular carcinoma (HCC) and glioblastoma (GBM). Both indications have received Orphan Drug Designation (ODD) and Fast Track Designation from the U.S. FDA, raising expectations for early commercialization. For glioblastoma, the therapy has been approved for an Expanded Access Program (EAP) and is currently being administered to patients. For hepatocellular carcinoma, Rznomics is conducting clinical trials in combination with immune-checkpoint inhibitors through clinical collaborations with Roche and Celltrion, which provide the necessary therapeutic agents for the trials free of charge.Beyond RZ-001, the company maintains a diverse pipeline addressing high unmet medical needs, including RZ-003 for Alzheimer’s disease and RZ-004 for retinitis pigmentosa (RP), aiming to provide fundamental solutions for these conditions.Rznomics’ technological benefit has been recognized both domestically and globally. Last year, the company was designated as the first "National Strategic Technology Company" and a "National Strategic Technology Possessing and Managing Company" by the Ministry of Science and ICT (MSIT) and the Korea Institute of Science & Technology Evaluation and Planning (KISTEP). Based on these credentials, Rznomics is pursuing the newly established "Deep Tech Special Listing". Upon listing, it is poised to become the first company among those managed by the MSIT to list under this specialized track.In May, Rznomics validated its technological competitiveness and commercial potential by signing a strategic global licensing agreement with Eli Lilly and Company, valued at approximately KRW 1.9 trillion (USD 1.3 billion), to co-develop RNA editing therapies. This partnership is significant as it secures a first-mover advantage in the next-generation gene therapy market with proprietary RNA editing technology.Rznomics plans to utilize the funds raised through its KOSDAQ listing to: advancing its RNA editing platform & accelerating global clinical trials and commercialization of core pipeline assets. Given the high scalability of its technology platform across various diseases, the company intends to expand collaborations with global pharmaceutical leaders through licensing and joint development based on its original patents and core technologies."Rznomics is presenting a new paradigm in the market for intractable cancer and rare disease treatments, where unmet medical needs are high, based on the world's first Trans-splicing Ribozyme platform," said Dr. Seong-Wook Lee, CEO of Rznomics. "We are committed to establishing our technology as the next-generation standard of care and evolving into a global leader in gene therapy, offering hope to patients suffering from intractable diseases."Rznomics is offering 2,060,000 shares for public subscription. The indicative price range is set between KRW 17,000 and KRW 22,500, with a target fundraising amount of KRW 35.0 billion to KRW 46.4 billion. Demand forecasting for institutional investors will take place from November 27 to December 3, followed by public subscription on December 9 and 10. Samsung Securities and NH Investment & Securities are serving as the joint underwriters.

2025-12-03
Rznomics to Participate in ‘BIO-Europe 2025’ to Expand Global Collaborations

Rznomics Inc. announced on the 3rd November that it will officially participate in BIO-Europe 2025, a premier global biopharma conference held in Vienna, Austria, from November 3 to 5. BIO-Europe is the largest partnering event in Europe, bringing together over 5,000 industry professionals from more than 60 countries.During the event, Rznomics plans to showcase its proprietary RNA editing platform and gene therapy development strategies. The company will actively seek opportunities for licensing agreements and co-development collaborations through strategic partnering meetings with global pharmaceutical companies, biotech firms, and investors.Rznomics has garnered significant attention within international academic and industrial circles for its differentiated technological platform: "Trans-splicing Ribozyme-based RNA Editing Therapeutics." In May, the company proved its technological innovation and global competitiveness by signing a research collaboration and licensing agreement with the U.S. pharmaceutical giant Eli Lilly and Company, with a total deal value reaching approximately KRW 1.9 trillion (USD 1.3 billion).As global interest in RNA-based therapeutic technologies continues to rise, Rznomics intends to discuss various collaboration models, including platform-based out-licensing and joint R&D, while further refining its plans for global market expansion through these high-level meetings."Since our major licensing deal with Eli Lilly, interest from leading global companies has surged significantly," said Sungwoo Hong, Vice President of Rznomics, who is attending the event. "Our participation in BIO-Europe 2025 will serve as a catalyst to accelerate additional licensing deals and expand our collaborative network with global industry leaders."

2025-11-04
Rznomics Receives Preliminary KOSDAQ Listing Approval

Rznomics Inc., a biotechnology company specializing in RNA-based gene therapeutics, officially announced on the 22nd Sep that it has received approval for its preliminary listing review from the Korea Exchange (KRX). With NH Investment & Securities and Samsung Securities serving as joint underwriters, the company is now initiating the full-scale listing process with the goal of completing its Initial Public Offering (IPO) by the end of the year.Founded in 2017, Rznomics develops innovative treatments for cancer and intractable rare diseases using its proprietary RNA replacement enzyme-based RNA editing and correction platform.Prior to this approval, Rznomics received "A" grades from two evaluation institutions designated by the KRX. Furthermore, the company was recognized as the first "National Strategic Technology Company" by the Ministry of Science and ICT (MSIT) and the Korea Institute of Science & Technology Evaluation and Planning (KISTEP) in May last year. In September, it was officially designated as a company possessing and managing National Strategic Technology.Leveraging these credentials, Rznomics is set to list via the "Deep Tech Special Listing". It is poised to become the first company among those managed by the MSIT to complete an IPO under this specialized track. Notably, the approval process was expedited, taking only 40 business days—five days shorter than the standard 45-day review period.Rznomics currently maintains a robust pipeline of core assets targeting intractable cancers, including hepatocellular carcinoma (HCC) and glioblastoma (GBM), as well as rare diseases such as Alzheimer’s, hereditary retinitis pigmentosa (RP), and rare neurological disorders. Its lead candidate, RZ-001, an oncology gene therapy, has significantly enhanced its commercialization potential by receiving both Fast Track Designation and Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA).In a major strategic milestone last May, Rznomics signed a global platform-based licensing agreement with Eli Lilly and Company to develop RNA editing therapies, validating the global competitiveness and commercial viability of its platform technology.As domestic and international pharmaceutical companies increasingly seek new modalities—such as ADCs, Targeted Protein Degraders (TPD), and Cell and Gene Therapies (CGT)—Rznomics is expected to achieve rapid growth through further partnerships with global biopharma leaders."Rznomics is developing innovative therapeutics that overcome existing limitations based on the world’s first Trans-splicing Ribozyme platform," said Dr. Seong-Wook Lee, CEO of Rznomics. "Through this KOSDAQ listing, we will open a new paradigm for next-generation RNA editing therapies on the global stage."

2025-09-22
Rznomics’ Anti-cancer Gene Therapy, RZ-001, Selected for National New Drug Development Project Supporting Clinical Development

Rznomics, a biopharmaceutical company specializing in RNA-based gene therapies (CEO Seong-wook Lee), announced today that its lead anti-cancer candidate, RZ-001, has been selected as a clinical-stage project for the “2025 1st National New Drug Development Project” by the Korea Drug Development Fund (KDDF).The National New Drug Development Project is a pan-governmental R&D initiative aimed at strengthening the global competitiveness of the Korean pharmaceutical and biotech industries. Following this selection, Rznomics will receive R&D funding for the next two years to support the clinical development of RZ-001 for the treatment of Glioblastoma (GBM).Glioblastoma is the most aggressive and fatal form of glioma, an intractable brain tumor with a five-year survival rate of less than 10%. As the most common primary brain tumor in adults, it affects approximately 3 to 4 per 100,000 people. Despite standard-of-care treatments—including surgery, chemotherapy, and radiation therapy—the recurrence rate remains high, representing a significant unmet medical need. Notably, the field has seen a lack of effective new therapies for over a decade, creating an urgent demand for treatments with innovative mechanisms of action.RZ-001 is a next-generation gene therapy that specifically targets and cleaves human telomerase reverse transcriptase (hTERT) mRNA, which is overexpressed in cancer cells. It then replaces the targeted sequence with a therapeutic RNA to induce selective apoptosis (cell death) of the cancer cells. This process is powered by Rznomics’ proprietary RNA trans-splicing ribozyme technology.Currently, RZ-001 is undergoing Phase 1 clinical trials for GBM in South Korea. With the recent Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA), Rznomics expects to accelerate its global clinical development.The innovation and safety profile of RZ-001 have been recognized by the U.S. FDA through both Orphan Drug Designation (ODD) and Fast Track status. Furthermore, RZ-001 is being administered under an Expanded Access Program (EAP), which allows the use of investigational drugs for patients with serious conditions who have no comparable alternative therapies. This ongoing clinical experience is anticipated to have a positive impact on patient recruitment for upcoming clinical trials in the United States.“The selection for this national project will serve as a powerful catalyst for the global clinical advancement of RZ-001,” said Seong-wook Lee, CEO of Rznomics. “Leveraging our long-standing research expertise in trans-splicing ribozymes, we are dedicated to proving the potential of RZ-001 as a First-in-Class anti-cancer gene therapy.”

2025-06-20
Rznomics Passed Technology Evaluation, Advancing Toward KOSDAQ Listing

- Received “A, A” ratings from two technology evaluation institutions designated by the Korea Exchange- Plans to submit preliminary review application for KOSDAQ listing in the second half of the yearRznomics, a developer of RNA-based gene therapies, announced on June 11 that it has passed the technology evaluation required for a technology-special listing on the KOSDAQ market.The company received “A” grades from two technology evaluation institutions designated by the Korea Exchange (KRX), recognizing both its technological excellence and business potential. Rznomics plans to leverage the Deep Tech Track of the technology-special listing system starting in the second half of the year, with the goal of completing its KOSDAQ listing in the first half of next year.The technology evaluation is a mandatory step for the technology-special listing and assesses technological maturity, competitiveness, commercialization potential, and marketability. Under the Deep Tech Track—a newly introduced fast-track system—companies based on advanced science and national strategic technologies can apply for preliminary listing review with a single technology evaluation.In May 2024, Rznomics was recognized as the "National Strategic Technology Company No. 1" by the Ministry of Science and ICT (MSIT) and the Korea Institute of Science and Technology Planning and Evaluation (KISTEP). Later in September, the company was designated as a "National Strategic Technology Possession and Management Company," thereby qualifying for the Deep Tech Track. If successfully listed through this route, Rznomics would become the first company designated under MSIT’s national strategic technology program to achieve listing via the deep tech special track.Founded in 2017, Rznomics is developing anti-cancer and rare disease therapies using its proprietary RNA editing and correction platform based on RNA replacement enzymes. Founder and CEO Dr. Seong-Wook Lee began his RNA replacement enzyme research at Duke University Medical Center in the U.S., and has continued this work for over 20 years as a professor at Dankook University since 1997.Currently, the company is conducting Phase 1b/2 and Phase 1/2a clinical trials in South Korea and the United States for “RZ-001,” a gene therapy targeting hepatocellular carcinoma (HCC) and glioblastoma. Both indications have received Orphan Drug Designation (ODD) and Fast Track designation from the U.S. Food and Drug Administration (FDA). For glioblastoma, the therapy is also being provided to patients under an Expanded Access Program (EAP) due to the lack of alternative treatments. In addition, “RZ-004,” a therapy for hereditary retinitis pigmentosa, has received approval for a Phase 1 trial in Australia, with patient enrollment expected to begin soon.In May, Rznomics signed a strategic global licensing agreement with U.S. pharmaceutical company Eli Lilly and Company to co-develop RNA editing therapies, further validating the global competitiveness and commercial potential of its platform technology. The company is also expanding its scope beyond RNA replacement enzymes to include circular RNA platforms and is collaborating with various companies on vaccine and therapeutic development and validation.Despite a challenging biotech investment environment in the second half of last year, Rznomics successfully raised KRW 20.3 billion (around USD 15M) in a Pre-IPO Series round from investors including KB Investment, Aon Investment, Yuanta Investment, Samsung Venture Investment, and Quad Ventures, demonstrating its strong market potential.CEO Dr. Seong-Wook Lee commented, “Passing the technology evaluation marks official recognition of Rznomics’ proprietary RNA-based platform and its growth potential,” adding, “Through our KOSDAQ listing, we aim to establish a solid foundation for business expansion and become a global leader in RNA editing.”

2025-06-11
Rznomics Enters Global Licensing Agreement with Eli Lilly for RNA-editing Therapeutics

Co-development of precision RNA therapeutics for inherited hearing loss treatmentRznomics Inc., a South Korea-based biopharmaceutical company specializing in RNA-based therapeutics, announced today that it has entered into a strategic global research collaboration and licensing agreement with Eli Lilly and Company (NYSE: LLY) to develop and commercialize novel RNA-editing therapies using Rznomics' proprietary trans-splicing ribozyme platform.The collaboration focuses on the discovery and development of RNA-editing therapeutics for sensorineural hearing loss. Rznomics will conduct early-stage research according to the jointly approved research plans, while Lilly will assume responsibility for further development and commercialization.If Lilly exercises all available options under the agreement, the total deal value could reach more than $1.3 billion, as well as separate royalties on product sales. In line with the mutual understanding between the two companies, the upfront payment was not disclosed. “We are thrilled to collaborate with Lilly, a global leader in pharmaceutical innovation,” said Dr. Seong-Wook Lee, CEO of Rznomics. “This partnership validates our trans-splicing ribozyme platform and opens the door to treating previously intractable diseases with precision RNA therapeutics. Together with Lilly, we aim to accelerate the development of transformative therapies for patients around the world.”This partnership marks a significant milestone for Rznomics as it seeks to expand its presence in the global biotech arena. For Lilly, the deal aligns with its broader strategy to build a leading RNA therapeutics pipeline and address high unmet needs in hearing loss and other therapeutic areas.About Rznomics 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.

2025-05-15

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