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, targeting cholestatic diseases [1][2] - AX-0810 is the first investigational RNA editing oligonucleotide (EON) from ProQR's Axiomer™ platform, aimed at reducing toxic bile acid accumulation in the liver [6][10] Company Overview - ProQR Therapeutics is focused on developing transformative RNA therapies using its proprietary Axiomer technology, which utilizes the body's own ADAR enzymes for RNA editing [1][10] - The company aims to create a new class of medicines for both rare and prevalent diseases with unmet medical needs [10] 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][6] - The trial will be conducted in the Netherlands, and dosing is set to begin following the CTA approval [2] Upcoming Events - ProQR will host a virtual Investor and Analyst Event on November 3, 2025, to discuss the clinical development of AX-0810 [2][8] - The event will feature presentations from ProQR management and Professor Henkjan J. Verkade, a key opinion leader in pediatric gastroenterology [2][4]