ProQR Therapeutics N.V.
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- ProQR Therapeutics N.V. (Nasdaq: PRQR) is a clinical-stage biotechnology company that discovers, develops and advances RNA therapies based on its proprietary Axiomer RNA editing platform technology, which leverages ADAR-mediated editing to correct single nucleotide mutations or modulate protein expression in RNA for rare and prevalent diseases with high unmet need. Founded in 2012 and headquartered in Leiden, the Netherlands, with operations extending to Cambridge, Massachusetts, the company targets disorders originating primarily in the liver and nervous system through programs deliverable to hepatocytes; its main products in the pipeline include AX-0810 for cholestatic diseases targeting Na-taurocholate cotransporting polypeptide (NTCP), AX-1412 for cardiovascular diseases targeting Beta-1,4-galactosyltransferase 1 (B4GALT1), AX-2402 for Rett syndrome, AX-2911 for metabolic dysfunction-associated steatohepatitis (MASH) targeting patatin-like phospholipase domain-containing protein 3 (PNPLA3), AX-0601 for obesity and Type 2 diabetes, AX-1005 for undisclosed cardiovascular disease targets, AX-9115 for a rare metabolic condition, and additional early-stage efforts in central nervous system disorders and other unnamed targets. ProQR maintains strategic collaborations including a global licensing and research partnership with Eli Lilly and Company since 2021—expanded in December 2022 from five to ten targets with an option for fifteen, yielding increased revenue from new projects in 2024—a September 2024 partnership with Théa Open Innovation to further develop sepofarsen and ultevursen for rare genetic eye diseases, and an expanded $9.1 million funding agreement with the Rett Syndrome Research Trust announced in December 2024 to accelerate AX-2402 toward clinical trials, alongside anticipated mid-2025 updates on GalNAc delivery optimization and potential milestone income from existing alliances.
Company News
ProQR Therapeutics N.V. (NASDAQ:PRQR) Stock Has Consensus Price Target of $7.60 According to Analysts
ProQR Reports First Human Validation of RNA Editing Platform in Liver Disease
ProQR Therapeutics N.V. (PRQR) Shareholder/Analyst Call Transcript
ProQR Announces Appointment of Chris Hart as Chief Data and AI Officer and Thomas Wolf to join the Board of Directors as Strategic AI Advisor
ProQR Announces First Participant Dosed in Phase 1 Study of AX-0811 and Virtual Investor and Analyst Educational Event on September 30, 2026
ProQR to Participate in Upcoming Investor Conferences in September 2026
ProQR Therapeutics: Buy For Axiomer RNA Tech Validation And Other Catalysts
ProQR Announces Second Quarter 2026 Operating and Financial Results
Contrasting Artiva Biotherapeutics (NASDAQ:ARTV) & ProQR Therapeutics (NASDAQ:PRQR)
ProQR Therapeutics N.V. (NASDAQ:PRQR) Receives Average Rating of “Moderate Buy” from Analysts
ProQR Therapeutics: Human NTCP Data Strengthens The Axiomer Thesis
ProQR Prices $50.0 Million Underwritten Registered Direct Offering and Concurrent Private Placement
ProQR Announces Positive Phase 1 Target Engagement Data for AX-0810, Establishing First Clinical Validation of the Axiomer RNA Editing Platform
ProQR Announces Positive Phase 1 Target Engagement Data for AX-0810, Establishing First Clinical Validation of the Axiomer RNA Editing Platform
ProQR Announces First Quarter 2026 Operating and Financial Results
ProQR Announces Multiple Upcoming Scientific Conference Presentations Highlighting the Breadth of its Axiomer™ RNA Editing Platform
Earnings Preview: ProQR (PRQR) Q1 Earnings Expected to Decline
ProQR Announces Annual General Meeting of Shareholders to be Held June 2, 2026
ProQR Announces Nomination of Dr. Lykke Hinsch Gylvin to its Board of Directors
ProQR Therapeutics Unveils New RNA Editing Programs; AX-0810 Phase 1 Target Data Due This Quarter