ProQR Therapeutics Receives CTA Authorization for AX-0810 and Announces Investor Event

ProQR Therapeutics N.V. announced that it has received authorization from the Central Committee on Research Involving Human Subjects (CCMO) for its Clinical Trial Application (CTA) for AX-0810, a Phase 1 study targeting NTCP for the treatment of cholestatic diseases. The study will evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers. ProQR also announced a virtual Investor and Analyst Event on November 3, 2025, to discuss the clinical development of AX-0810. The event will feature presentations by ProQR management and Key Opinion Leader Professor Henkjan J. Verkade, MD, PhD. AX-0810 is a first-in-class investigational RNA editing oligonucleotide (EON) that aims to reduce toxic bile acid accumulation in the liver, potentially offering a disease-modifying treatment for cholestatic diseases.