Gene Therapy-Based Cancer Drug Candidate RZ-001 Gains Fast Track Status for Hepatocellular Carcinoma, Following Glioblastoma

Rznomics announced on the 17th that its gene therapy-based cancer drug candidate, RZ-001, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of hepatocellular carcinoma.

This is the second Fast Track designation for RZ-001, following its 2023 designation for glioblastoma, an aggressive and hard-to-treat brain cancer.

Fast Track is an FDA program designed to expedite the development and review of drugs intended for serious or life-threatening conditions. It allows companies to have more frequent communication with the FDA, benefit from priority review, and submit their application for rolling review—where different sections of the application are reviewed as they become available, rather than waiting for the full submission—potentially speeding up the approval process.

RZ-001 is being developed using Rznomics' proprietary RNA editing and correction platform, which is based on ribonucleic acid substitution enzymes. The company is currently conducting Phase 1b/2a clinical trials for hepatocellular carcinoma and Phase 1/2a trials for glioblastoma in South Korea, with FDA approval to conduct the same trials in the United States.

For glioblastoma, Rznomics has also received FDA approval for an Expanded Access Program (EAP), allowing compassionate use of RZ-001. This program is currently being conducted at Harvard University Hospital.

Seong-wook Lee, CEO of Rznomics, stated, "This Fast Track designation reaffirms the potential of RZ-001 as an innovative cancer treatment. We are committed to accelerating clinical development to provide effective treatment options for patients suffering from difficult-to-treat cancers."