ProQR highlights key updates from presentation of Axiomer RNA platform

ProQR Therapeutics highlighted key updates from its oral presentation at the 5th International Conference on Base Editing, Prime Editing & Related Enzymes in San Diego, California. “We were pleased to present new in vivo data for our proprietary Axiomer RNA editing technology platform at the Deaminet 2024 meeting, demonstrating robust editing of ACTB in the liver of NHP, as well as functional protein data with the liver target ANGPTL3 in mice,” said Gerard Platenburg, Chief Scientific Officer of ProQR. “To date, we have generated robust in vitro and in vivo preclinical platform data across a range of liver and CNS targets. These encouraging data and the progress of our science reinforce the potential of our Axiomer RNA editing oligonucleotides, and we look forward to presenting additional platform data, as well as the first preclinical data for our pipeline programs at upcoming scientific meetings, as we move our technology forward towards trials in humans.” Key updates from the presentation included: Axiomer Editing Oligonucleotides demonstrated high intrinsic editing capability in the liver across models. Presentation of new data highlighting up to 70% editing efficiency of ACTB in liver of mice and NHPs at multiple timepoints. Editing levels confirmed through several reliable RNA analysis methods, including dPCR and RNAseq, showing consistent results. An EON editing ANGPTL3 in mice resulted in a 2-fold increase in lipoprotein lipase activity and a related decrease of LDL cholesterol in plasma of 40% in vivo. Preliminary nonclinical safety assessment showed a similar safety profile compared to other single-stranded RNA oligonucleotides. Ongoing platform advancements anticipated through increased knowledge of ADAR biology, selection of models for prediction of Axiomer potential in human tissue, EON sequence optimization, and investigation of delivery options.