Presented at the RNA Editing Gordon Research Conference on March 23, 2023 by Gerard Platenburg - ProQR Therapeutics
Unlocking the Potential of Innovative Editing Oligonucleotides to Address Liver Originated Disorders
Presentation
Gepubliceerd op op RNA Editing Gordon Research Conference
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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.
Gerard Platenburg - Chief Scientific Officer and co-founder
Gerard is a co-founder and has served as our Chief Scientific Officer since 2022, following his tenure as Chief Innovation Officer from 2014 to 2022. Gerard has an extensive background in RNA modulation and orphan drug discovery and development and is currently in charge of our Innovation unit.
