ProQR Therapeutics announced several presentations highlighting the Company’s proprietary Axiomer RNA editing technology platform at upcoming scientific and industry conferences, including the 8th annual Oligonucleotide and Precision Therapeutics Congress, March 13-14, 2023 in Boston, MA, U.S.; RNA Leaders Europe Congress, March 15-16, 2023, in Basel, Switzerland; and the bi-annual RNA Editing Gordon Research Conference, March 19-24, 2023 in Ventura, CA, U.S. Presentation and panel session at the Oligonucleotide and Precision Therapeutics Congress: Oral presentation title: Therapeutic potential of an RNA editing platform using Editing Oligonucleotides: Abstract: Axiomer, an RNA editing technology, uses Editing Oligonucleotides to target specific RNA for A-to-I editing by recruiting endogenous Adenosine Deaminase Acting on RNA. This presentation will highlight the therapeutic possibilities of this platform that are not limited to disease causing mutations and can potentially address high unmet medical needs by editing wild-type RNA to engineer proteins or modify their function as well as creating de novo mutations. Panel session title: Integrating Innovative Technologies into Oligo Therapeutics Discovery and Delivery. Presentation at RNA Leaders Europe Congress: Oral presentation title: Unlocking the potential of Axiomer platform to develop transformative RNA editing therapies : Abstract: The presentation will describe: Axiomer, the next-generation RNA technology using the cell’s own editing machinery; explore therapeutic opportunities of Axiomer, beyond disease-causing mutations; and the advancement of Axiomer through in-house pipeline programs and partnering strategy. Presentation at the RNA Editing Gordon Research Conference:Oral presentation title: Development of RNA editing oligonucleotides technology to address liver originated disorders: Abstract: Axiomer, an RNA editing platform, uses chemically modified and complementary single stranded editing oligonucleotides to target specific RNA for A-to-I editing by recruiting endogenous Adenosine Deaminase Acting on RNA. 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.
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