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ProQR Receives CTA Authorization for AX-0810 and Announces Virtual Investor and Analyst Event on November 3, 2025
LEIDEN, Netherlands & CAMBRIDGE, Mass., Oct. 20, 2025 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing
About this update from Proqr Therapeutics N.v.
[{"type":"text","content":"LEIDEN, Netherlands & CAMBRIDGE, Mass., Oct. 20, 2025 (GLOBE NEWSWIRE) -- 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. In connection with this milestone, ProQR will host a virtual Investor and Analyst Event entitled: “Entering the Clinic with AX-0810: Establishing Safety, PK, and the Biomarker Roadmap for Proof of Target Engagement” The event will take place on November 3, 2025 beginning at 10 am ET and will feature presentations by ProQR Management and Key Opinion Leader Professor Henkjan J. Verkade, MD, PhD, a pediatric gastro/hepatologist at the Beatrix Children’s Hospital of the University Medical Center Groningen. Event Details and Registration Title: Entering the Clinic with AX-0810: Establishing Safety, PK, and the Biomarker Roadmap for Proof of Target EngagementDate & Time: November 3, 2025 beginning at 10 am ET until approximately 11:30 am ETVirtual format: Webcast presentations and Q&A with covering analysts and ManagementRegistration: https://lifescievents.com/event/agp29t7j/ KOL Biography – Prof. dr. Henkjan J. Verkade, MD, PhD Professor Henkjan J. Verkade, MD, PhD is a pediatric gastro/hepatologist at the Beatrix Children’s Hospital of the University Medical Center Groningen, The Netherlands. He received his PhD degree in Medicine cum laude at the University of Groningen on the thesis entitled “Lipid absorption and metabolism”. He was a post-doctoral fellow at the University of Alberta, Edmonton, Ca...