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

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  • 날짜
    2025-06-20
기사원문
Rznomics' gene therapy selected for Korea's national drug development project
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.”
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