en.Wedoany.com Reported - Rznomics' RNA editing-based anticancer gene therapy, RZ-001 (Taspitimagene advec), has demonstrated manageable safety and preliminary efficacy in a Phase 1/2a clinical trial for recurrent glioblastoma (rGBM), with multiple patients achieving long-term tumor recurrence suppression and disease control for over six months. These interim results were presented on June 13, 2026, at the Asian Society for Neuro-Oncology Annual Meeting (ASNO 2026) held in Kanazawa, Japan.
Dr. Chae-Yong Kim, a professor in the Department of Neurosurgery at Seoul National University Bundang Hospital, delivered an oral presentation at the conference. RZ-001 is designed using Rznomics' proprietary RNA trans-splicing ribozyme platform, which enables precise RNA editing to allow the drug to specifically express therapeutic genes within tumor cells, inducing cancer cell death. Headquartered in South Korea, the company is a clinical-stage biopharmaceutical firm focused on developing RNA gene therapies with broad applicability across multiple indications.
As of the data cutoff, a total of 20 patients had completed screening, of whom 10 were formally enrolled and treated. The interim analysis revealed no new or unexpected treatment-related safety issues, no dose-limiting toxicities (DLTs) were reported, and no treatment-related adverse events of Grade 4 or higher were observed. Most adverse events were considered related to the disease or typical underlying conditions in patients with glioblastoma.
Recurrent glioblastoma is one of the most aggressive and difficult-to-treat malignant brain tumors, with high recurrence rates even after standard therapy and a very poor prognosis. Dr. Chae-Yong Kim stated that the data obtained so far indicate that RZ-001 has a manageable safety profile while showing encouraging signs of preliminary clinical activity. He added that, given that recurrent glioblastoma typically recurs within two to four months, the observed long-term recurrence suppression—particularly data from patients exceeding nine months—is very encouraging, and he looks forward to further clarifying its full clinical value through additional patient enrollment and long-term follow-up.
RZ-001 is also under clinical development for hepatocellular carcinoma (HCC), a program that received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA in May 2026. The company signed a research collaboration and licensing agreement with global pharmaceutical company Eli Lilly in May 2025 and was listed on the KOSDAQ market in December 2025.
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