ProQR Therapeutics N.V.
(NASDAQ:PRQR) on Thursday reported positive early-stage clinical data for its investigational RNA editing therapy AX-0810, while simultaneously announcing a $50 million underwritten offering aimed at supporting development of its pipeline and broader corporate operations.
AX-0810 Demonstrates Strong Early Activity In Phase 1 Trial The clinical-stage biotechnology company said findings from its ongoing Phase 1 multiple ascending dose study showed evidence of target engagement for AX-0810, its first investigational Axiomer RNA editing oligonucleotide, in healthy volunteers.
AX-0810 is designed to selectively modulate NTCP, a transporter involved in bile acid uptake into the liver.
Read Also: Korro Bio Stock Gains After Tough 2025 – New Drug Candidate, Analyst Upgrades Boost Outlook The therapy aims to reduce toxic bile acid buildup associated with cholestatic liver diseases, including biliary atresia.
