An Innovative Solution for Modern Functional Genomics and Target Validation
Advirna’s self-deliverable RNAi technology is based on the ability of chemically modified RNA molecules to penetrate cells and deliver specific gene knockout by RNAi mechanism. Self-deliverable RNA molecules do not require any transfection reagent, vehicle or special cell treatment.
Self-deliverable RNAs induce specific gene knockout in vitro in any cell type tested, including hard-to-transfect cell lines, primary cells, suspension cultures. No cellular toxicity is observed.
No Delivery Vehicle Required
Self-deliverable RNAs are efficient in ex-vivo tissue models and in vivo. The first drug candidate based on self-deliverable RNAi technology developed by RXi Pharmaceuticals is currently in Phase II Clinical Trials for multiple indications.
The primary areas of the use of self-deliverable RNAi technology are hard-to-transfect cell lines, in vivo and ex-vivo target validation, in vivo functional genomics and biomanufacturing. The functional properties of sdRNAs are summarized in the table below.