- ProQR Therapeutics N.V. has received **Clinical Trial Application (CTA) authorization** from the Central Committee on Research Involving Human Subjects (CCMO) for a **Phase 1 study of AX-0810** in healthy volunteers.  **[1612940_28f18813826d4f69bbb05a7b877ece1c_0]** 
- This authorization allows ProQR to begin dosing in the Phase 1 study, which will evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics via biomarkers to establish proof of target engagement.  **[1612940_28f18813826d4f69bbb05a7b877ece1c_0]** 
- AX-0810 is ProQR's lead investigational RNA editing oligonucleotide (EON) targeting NTCP, developed for the treatment of **cholestatic diseases** like primary sclerosing cholangitis and biliary atresia.  **[1612940_28f18813826d4f69bbb05a7b877ece1c_0]** 
- ProQR will host a virtual **Investor and Analyst Event on November 3, 2025, at 10 am ET**, to discuss entering the clinic with AX-0810.  **[1612940_28f18813826d4f69bbb05a7b877ece1c_0]** 
- The company anticipates the **timing of initial Phase 1 clinical data for AX-0810 in Q4 2025**.  **[1612940_28f18813826d4f69bbb05a7b877ece1c_3]**

ProQR Therapeutics (PRQR)