RZ-001

RZ-001 is an investigational RNA-based gene therapy developed for the treatment of cancers such as hepatocellular carcinoma (HCC) and glioblastoma multiforme (GBM). It utilizes a viral vector to deliver a proprietary trans-splicing ribozyme that targets and cleaves human telomerase reverse transcriptase (hTERT) mRNA in cancer cells. This process downregulates hTERT expression and replaces it with a therapeutic gene sequence, specifically herpes simplex virus thymidine kinase (HSV-TK), leading to direct cytotoxicity in tumor cells. Additionally, RZ-001 modulates the tumor microenvironment by reducing vascular endothelial growth factor (VEGF) expression and promoting immune cell infiltration, thereby enhancing responsiveness to immunotherapy. The therapy is being evaluated both as monotherapy and in combination with agents such as valganciclovir, atezolizumab, and bevacizumab[2][3][4][5][6][9].

Other names

hTERT-targeting trans-splicing ribozymeAd-hTERT-TR-HSVtk