Rznomics Receives WHO INN Designation for RNA-Based Anticancer Gene Therapy RZ-001

May 27 , 2026
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May 27, 2026 —

Rznomics announced that the World Health Organization has assigned the International Nonproprietary Name “Taspitimagene advec” to its RNA-based anticancer gene therapy candidate RZ-001. The name was selected by the WHO INN Expert Advisory Panel under the latest naming conventions for gene therapies and is expected to be listed as a Proposed INN in the upcoming WHO Drug Information List 136, scheduled for publication in 2026.

The INN designation marks an important step in RZ-001’s transition from an internal development code to an internationally recognized product name used in global regulatory, clinical, academic, and commercial communication. For gene therapy developers, an INN can help support clearer identification of a product across jurisdictions and facilitate future regulatory review and licensing discussions.

Taspitimagene advec is a first-in-class anticancer gene therapy based on Rznomics’ proprietary trans-splicing ribozyme platform. The therapy is designed to selectively express therapeutic genes in cancer cells, inducing tumor cell death while minimizing effects on normal tissues. This RNA-based mechanism differentiates the program from more conventional viral gene addition or genome-editing approaches.

Rznomics is currently conducting clinical trials in glioblastoma and hepatocellular carcinoma, following IND approvals from both Korea’s Ministry of Food and Drug Safety and the U.S. FDA. The company previously presented interim Phase 1/2a monotherapy data in glioblastoma at ESMO Asia and later disclosed safety and preliminary efficacy results from a Phase 1b/2a study evaluating Taspitimagene advec in combination with atezolizumab and bevacizumab for hepatocellular carcinoma at the AACR Annual Meeting.

The program has also received multiple U.S. FDA designations for hepatocellular carcinoma, including Orphan Drug Designation, Fast Track designation, and Regenerative Medicine Advanced Therapy designation. These designations highlight the potential clinical relevance of Taspitimagene advec as a novel therapeutic option for liver cancer.

Rznomics said the WHO INN designation is expected to support the company’s global clinical development, regulatory strategy, and commercialization planning as it continues to advance its RNA-based gene therapy platform across oncology indications.

Source:

https://www.koreabiomed.com/news/articleView.html?idxno=31793

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