ProQR Therapeutics Gets European Medicines Agency's Committee's Authorization For Clinical Trial Application For Phase 1 Trial Of AX-0810 To Treat Clerosing Cholangitis, Biliary Atresia

Author: Benzinga Newsdesk | October 20, 2025 07:12am

ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced that following review under the new European Medicines Agency (EMA) centralized review process, the Central Committee on Research Involving Human Subjects (CCMO) has authorized ProQR's Clinical Trial Application (CTA) for a Phase 1 study of AX-0810 in healthy volunteers. AX-0810 is the Company's lead investigational editing oligonucleotide (EON) targeting NTCP, which is being developed for the treatment of cholestatic diseases like primary sclerosing cholangitis and biliary atresia.

With this CTA approval, ProQR is authorized to begin dosing in its Phase 1 study, which is being conducted in the Netherlands. The Phase 1 study will evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) via biomarkers to establish proof of target engagement.

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