ProQR Receives EMA Authorization to Begin Phase 1 Trial of AX-0810 RNA Editing Therapy for Cholestatic Diseases
- ProQR Therapeutics received authorization from the Central Committee on Research Involving Human Subjects (CCMO) following EMA centralized review to initiate a Phase 1 study of AX-0810 in healthy volunteers.
- AX-0810 is a first-in-class investigational RNA editing oligonucleotide targeting NTCP, developed for treating cholestatic diseases like primary sclerosing cholangitis and biliary atresia.
- The Phase 1 study will evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics via biomarkers to establish proof of target engagement in the Netherlands.
- AX-0810 represents the first program from ProQR's Axiomer RNA editing pipeline to enter clinical development, with initial clinical data expected in Q4 2025.
