Abstract

Silence Therapeutics is a clinical stage biopharma company developing therapeutic siRNAs for a range of indications with therapeutic targets originating in hepatocytes. Across the field there are today five approved therapeutic siRNAs and many additional ones in development, most of them also targeted to hepatocytes. The field is maturing, however there are still pre-clinical challenges remaining for finding the most suitable therapeutic siRNAs when we move through the drug discovery funnel. I will share some examples of such challenges and the methods we are validating with the intent to generate data that can help solving them.