ProQR Announces Upcoming Presentation at RNA Editing Summit

Core Insights - ProQR Therapeutics is participating in the RNA Editing Summit from July 29-31, 2025, in Boston, MA, showcasing its advancements in RNA therapies using the Axiomer™ RNA editing technology platform [1][2] Company Overview - ProQR Therapeutics is focused on developing transformative RNA therapies, utilizing its proprietary Axiomer™ technology to create a new class of medicines for various diseases, including rare and prevalent conditions [5] - The Axiomer™ technology employs the cell's own ADAR machinery to make precise single nucleotide edits in RNA, potentially reversing mutations or modulating protein expression [4][5] Recent Developments - The company has submitted a Clinical Trial Application (CTA) for AX-0810, aimed at treating cholestatic diseases, and is making progress in central nervous system (CNS) applications of its Axiomer technology [2] - At the RNA Editing Summit, ProQR will present preclinical data related to its CNS programs and the Rett syndrome program targeting MECP2, highlighting the potential of Axiomer in addressing severe neurodevelopmental diseases [2][8] Future Directions - The presentation at the summit will include long-term and multiple dose data from non-human primate (NHP) studies, emphasizing the therapeutic applications of Axiomer in both CNS and liver diseases [8] - ProQR is committed to expanding its pipeline with a focus on unmet medical needs, aiming to advance its lead development programs and clinical trials in the coming years [9]