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ProQR Therapeutics NV. (Nasdaq: PRQR) reported Q1 2024 financial results, highlighting preclinical data for the AX-0810 program targeting NTCP for cholestatic diseases. The company presented successful defense of its IP estate and announced Martin Maier, PhD's nomination to the Board. ProQR emphasized its strong cash position of €102.7 million providing runway into mid-2026.
ProQR Therapeutics NV announced preclinical proof of concept data for its AX-0810 Axiomer™ RNA editing program targeting NTCP for cholestatic diseases. The data, presented at the ASGCT Annual Meeting, revealed significant progress in RNA editing technology. The AX-0810 program aims to reduce bile acid concentration in the liver by targeting NTCP, representing a promising approach for cholestatic disorders. The company plans to advance the program to the clinic in late 2024/early 2025, with additional milestones expected for the Axiomer pipeline.