ProQR Therapeutics NV Submits Clinical Trial Application for First Human Study of AX-0810 Targeting NTCP, Marking Milestone in Axiomer™ RNA Editing Platform Development

Reuters06-27

ProQR <a href="https://laohu8.com/S/LENZ">Therapeutics</a> NV Submits Clinical Trial Application for First Human Study of AX-0810 Targeting NTCP, Marking Milestone in Axiomer™ RNA Editing Platform Development

ProQR Therapeutics NV has announced the submission of a Clinical Trial Application $(CTA.UK)$ to the European Medicines Agency $(EMA)$ for their lead pipeline program, AX-0810, marking a significant advancement for their Axiomer™ RNA editing platform. This first-in-human Phase 1 trial will assess the safety, tolerability, pharmacokinetics, and target engagement of AX-0810 in healthy volunteers. AX-0810 targets NTCP, a liver cell protein, to potentially reduce toxic bile acid accumulation and address cholestatic liver diseases. The trial is set to begin at a single site in the Netherlands, with initial data expected in Q4 2025.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. ProQR Therapeutics NV published the original content used to generate this news brief via GlobeNewswire (Ref. ID: GNW1001114982-en) on June 26, 2025, and is solely responsible for the information contained therein.

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