RNA replacement enzyme vector-based cancer therapy (1st indication: Advanced HCC, RZ-001)

Mechanism of RNA replacement enzyme

Excellence	Specific regression of liver cancer cells expressing target RNAs & Specificity enhance by use of pro-drug Efficacy enhancement by suppressing target RNA expression & simultaneously triggering therapeutic gene expression in target cells Optimization of vector dose and minimization of side effects in non-tumor hepatocytes via miRNA regulation

In vivo Toxicity

Hepatocellular toxicity after injection of RZ-001: AST and ALT levels on days 15 and 29 after single intravenous injection of RZ-001 into normal mice

• Without GCV treatment

  

  AST and ALT levels of 0.5x10^10 and 1x10^10 VP-injected groups were observed to be at a level similar to those of a PBS-injected group.

• With GCV treatment

  

  Pro-drug, GCV, was administered for 10 days after injection of RZ-001.

  AST and ALT levels of 0.5x10^10 and 1x10^10 VP-injected groups were observed to be at a level similar to those of a PBS-injected group.

In vivo Efficacy

In vivo anti-cancer efficacies of hit and RZ-001: Higher anti-cancer efficacy than hit in Xenograft liver metastasis HCC animal model