Presenter: Gerard Platenburg, CSO and co-founder, ProQR Therapeutics
Date: Thursday, March 23
Abstract: Axiomer, an RNA editing platform, uses chemically modified and complementary single stranded editing oligonucleotides (EONs) to target specific RNA for A-to-I editing by recruiting endogenous Adenosine Deaminase Acting on RNA (ADAR). Through now further developed design principles there has been an established improvement of specificity and efficacity of EONs, and we have tested the RNA editing efficacy and short-term safety of EONs acting on various genes and models. The increasing knowledge on the platform have unlocked therapeutic applications beyond disease-causing mutations and the presentation will review the potential of Axiomer platform in multiple organs as well as its applicability for the treatment of liver originated disorders including data from preclinical models.
