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.
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