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ProQR Therapeutics (PRQR) is a clinical-stage biopharmaceutical company pioneering RNA-based therapies for severe genetic disorders. This page provides investors and industry observers with timely updates on clinical developments, regulatory milestones, and strategic partnerships related to its Axiomer RNA editing platform.
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Discover updates on therapeutic programs targeting conditions like cystic fibrosis and Rett syndrome, financial disclosures, and partnership announcements with industry leaders. All content is sourced from official channels and reputable financial publications to maintain accuracy and compliance.
Bookmark this page for streamlined access to PRQR's latest scientific advancements and corporate updates. Check regularly for new developments in RNA editing innovation and the company's progress toward delivering transformative genetic medicines.
ProQR Therapeutics NV (Nasdaq: PRQR) reported its Q2 2024 financial and operating results. Key highlights include:
- AX-0810 (for cholestatic diseases) and AX-1412 (for cardiovascular diseases) programs advancing to clinic in late 2024/early 2025
- $4.5 million in milestones achieved in H1 from Lilly partnership
- €96.2 million cash position as of Q2 end, providing runway into mid-2026
- Net loss of €2.7 million (€0.03 per diluted share) for Q2 2024
- R&D costs increased to €7.1 million in Q2 2024 from €5.9 million in Q2 2023
- General and administrative costs decreased to €3.0 million in Q2 2024 from €4.1 million in Q2 2023
ProQR plans to announce translational data and clinical trial designs for AX-0810 and AX-1412 in H2 2024.
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.