ProQR Therapeutics N.V. reports developments as a clinical-stage biopharmaceutical company built around its proprietary Axiomer™ RNA editing platform. The technology uses ADAR-mediated RNA editing to make single-nucleotide changes in RNA, with pipeline updates centered on AX-0810 and additional programs across rare and prevalent diseases with unmet need.
Recurring company news includes scientific conference presentations, investor and analyst events, clinical-program updates, development-candidate selections, AI-enabled discovery initiatives, and collaborations such as Ginkgo Bioworks and Eli Lilly. ProQR also reports annual operating and financial results, annual general meeting materials, board composition changes, and other governance matters tied to its Nasdaq-listed public-company profile.
ProQR Therapeutics (Nasdaq: PRQR) announced its participation in the RNA Editing Summit from June 18-20, 2024, in Boston. Gerard Platenburg, Chief Scientific Officer and Co-Founder, will present on June 20 at 2:00pm ET. The presentation, titled “Developing Axiomer RNA Editing Technology Towards Clinical Development,” will focus on ProQR’s ADAR-mediated Axiomer RNA editing technology, optimization strategies, and the AX-0810 program targeting NTCP for cholestatic diseases. The presentation will also cover in vivo models of biomarkers for NTCP and will be accessible on ProQR’s website under Presentations & Publications.
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.
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