ProQR Receives CTA Authorization for AX-0810 and Announces Virtual Investor and Analyst Event on November 3, 2025

Core Insights - ProQR Therapeutics has received authorization from the Central Committee on Research Involving Human Subjects (CCMO) for a Phase 1 Clinical Trial Application (CTA) for AX-0810, an investigational RNA editing oligonucleotide targeting NTCP, aimed at treating cholestatic diseases such as primary sclerosing cholangitis and biliary atresia [1][2] Company Overview - ProQR Therapeutics is focused on developing transformative RNA therapies using its proprietary Axiomer™ RNA editing technology platform, which utilizes the body's endogenous ADAR enzymes for RNA editing [1][8] - The company aims to create a new class of medicines for both rare and prevalent diseases with unmet medical needs [8] Clinical Development - The Phase 1 study of AX-0810 will assess safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) through biomarkers to establish proof of target engagement [2][5] - AX-0810 is the first program from ProQR's Axiomer™ RNA editing pipeline to enter clinical development, with the trial being conducted in the Netherlands [2][5] Upcoming Events - ProQR will host a virtual Investor and Analyst Event on November 3, 2025, to discuss the clinical trial and present findings related to AX-0810 [2][6] - The event will feature presentations from ProQR management and Professor Henkjan J. Verkade, a key opinion leader in pediatric gastroenterology/hepatology [2][3] Research Focus - Professor Verkade's research interests include pediatric cholestatic liver diseases, mechanisms of bile acid transport, and the development of biomarkers and novel therapies for rare inherited liver disorders [4] - He has authored over 300 peer-reviewed publications, contributing significantly to the field [4] Technology Platform - Axiomer™ technology allows for specific single nucleotide changes in RNA, potentially correcting disease-causing mutations and modulating protein expression [6][7] - This innovative approach aims to reduce toxic bile acid accumulation in the liver, addressing the underlying causes of cholestatic diseases [5][7]