On October 20, 2025, ProQR Therapeutics N.V. announced that the Central Committee on Research Involving Human Subjects (CCMO) has authorized its Clinical Trial Application (CTA) for a Phase 1 study of AX‑0810 in healthy volunteers. The authorization was granted under the European Medicines Agency’s (EMA) new centralized review process, marking the first regulatory approval for the company’s Axiomer RNA‑editing platform.
AX‑0810 is an investigational editing oligonucleotide that targets the sodium taurocholate cotransporting polypeptide (NTCP) to treat cholestatic diseases such as primary sclerosing cholangitis and biliary atresia. The Phase 1 study, conducted in the Netherlands, will evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics through biomarker analysis to establish proof of target engagement.
The CTA approval enables ProQR to begin dosing and collect early clinical data, a critical milestone that brings the company closer to demonstrating the clinical potential of its RNA‑editing technology. The company also announced a virtual investor and analyst event scheduled for November 3, 2025, to discuss the study’s design and the broader pipeline.
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