ProQR Therapeutics N.V. announced the submission of a Clinical Trial Application (CTA) to the European Medicines Agency (EMA) for its lead pipeline program, AX-0810. This submission marks the first time the company's Axiomer RNA editing platform will advance into clinical development.
AX-0810 is an investigational ADAR-mediated RNA editing oligonucleotide designed to target NTCP, a liver cell protein, to treat cholestatic diseases. The therapy aims to modulate NTCP function to reduce toxic bile acid accumulation, potentially mitigating inflammation and liver damage.
The proposed Phase 1 study will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of AX-0810 in healthy adult volunteers. Pending regulatory clearance, the study is expected to commence in the Netherlands, with initial data anticipated in the fourth quarter of 2025.
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