The Nucleonics’s HBV clinical candidate (NUC B1000) is an expressed interfering RNA (eiRNA) based product, consisting of a plasmid DNA construct that encodes four short interfering RNA (siRNA) molecules, each under the control of an RNA polymerase III promoter. Each of the four siRNAs targets a different segment of the HBV genome, collectively leading to the potential destruction or elimination of all RNA species produced by HBV within an infected cell. The result is a potent antiviral effect designed for efficacy against all HBV genotypes, including drug resistant strains.
Joseph Kim, CEO and president of VGX Pharmaceuticals, said: “The Nucleonics trial marks the first time that a targeted RNAi therapeutic has been systemically delivered to patients, and VGX is pleased to have been selected to manufacture the product under cGMP to mark this important milestone in the RNAi area.”