Welcome to our dedicated page for Proqr Therapeuti news (Ticker: PRQR), a resource for investors and traders seeking the latest updates and insights on Proqr Therapeuti stock.
ProQR Therapeutics N.V. (Nasdaq: PRQR) generates a steady flow of news as a clinical-stage biopharmaceutical company advancing RNA editing therapies. Its updates center on the Axiomer RNA base editing platform and a pipeline of editing oligonucleotides designed to correct or modulate disease-related targets at the RNA level. News coverage on this page reflects company announcements, clinical milestones, financial updates, and scientific presentations related to its programs in liver, CNS, and other genetically defined diseases.
Investors and followers of PRQR can expect news about the progress of AX-0810, ProQR’s lead investigational RNA editing oligonucleotide targeting NTCP for cholestatic diseases. Recent releases describe the submission and authorization of a Clinical Trial Application, the initiation of a Phase 1 first-in-human study in healthy volunteers in the Netherlands, and early safety and pharmacokinetic observations from the first cohort. Updates also outline plans for target engagement readouts and the future inclusion of a patient cohort in the same trial.
News items also highlight earlier-stage pipeline programs such as AX-2402 for Rett syndrome (MECP2 R270X), AX-2911 for metabolic liver disease including MASH (PNPLA3 I148M), and AX-1412 for cardiovascular disease (B4GALT1). ProQR reports non-clinical proof-of-concept data, development candidate selections, and functional results in disease-relevant models. Financial press releases provide quarterly operating and financial results, cash and cash equivalents, collaboration revenue from Eli Lilly, and comments on cash runway.
Additional PRQR news covers participation in scientific and investor conferences, including ASGCT, the RNA Editing Summit, OTS, TIDES USA, and healthcare investor events. These communications feature presentations on ADAR-mediated RNA editing of NTCP, PNPLA3, and MECP2, as well as platform-focused talks on optimizing editing oligonucleotides for therapeutic use. Bookmark this page to follow ProQR’s disclosed clinical data, pipeline milestones, collaboration developments, and financial reporting as they are released through press announcements and SEC-furnished materials.
ProQR (Nasdaq: PRQR) reported Q3 2025 operating and financial results and a program update.
Key updates: CTA authorization received for Phase 1 AX-0810 (NTCP) and a first-in-human study in healthy volunteers is being initiated; initial safety, tolerability and PK data for Cohort 1 expected by year-end 2025 and target engagement data across cohorts expected in H1 2026. Cash and cash equivalents were approximately €106.9 million at September 30, 2025, providing runway into mid-2027. Nine‑month net loss widened to €33.3 million.
ProQR (Nasdaq: PRQR) announced that the Central Committee on Research Involving Human Subjects (CCMO) has authorized its Clinical Trial Application (CTA) for AX-0810, permitting dosing to begin in a Phase 1 study in the Netherlands.
The Phase 1 trial in healthy volunteers will evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) via biomarkers to establish proof of target engagement for AX-0810, an investigational editing oligonucleotide targeting NTCP for cholestatic diseases.
ProQR will host a virtual Investor and Analyst Event on November 3, 2025 at 10:00 am ET with management and KOL Prof. Henkjan J. Verkade; registration is via the provided webcast link.
ProQR (Nasdaq: PRQR) said company management will participate in multiple investor conferences in October 2025: H.C. Wainwright Genetic Medicines Virtual Conference (Oct 14-15), H.C. Wainwright Liver Disease Virtual Conference (Oct 21-22) and Chardan 9th Annual Genetic Medicines Conference (Oct 21).
Presentations for the virtual conferences will be available on demand beginning Oct 14 and Oct 21 at 7:00 AM ET respectively, with virtual 1x1 meetings on the second day. At Chardan on Oct 21 management will join an RNA Editing panel at 1:00 PM ET and hold 1x1 investor meetings. Webcast details and archived webcasts (available ~30 days) are available in the company Investor Events section.
ProQR Therapeutics (Nasdaq: PRQR) reported its Q2 2025 financial and operational results, highlighting significant progress in its RNA editing pipeline. The company has submitted a Clinical Trial Application for AX-0810, its lead program targeting NTCP for cholestatic diseases, with initial Phase 1 data expected in Q4 2025.
ProQR maintains a strong financial position with €119.8 million in cash, providing runway into mid-2027. The company reported a net loss of €22.3 million for H1 2025. Notable developments include advancing AX-2402 for Rett Syndrome and achieving $2.0 million in milestone payments from its Eli Lilly collaboration, with potential for additional milestone income and expansion options.
ProQR Therapeutics (Nasdaq: PRQR) announced its upcoming participation in the RNA Editing Summit from July 29-31, 2025, in Boston, MA. Chief Scientific Officer Gerard Platenburg will present on July 31 at 3:45 PM ET, focusing on the company's Axiomer™ RNA editing technology platform.
The presentation will highlight preclinical NHP data in CNS applications, including their Rett program targeting MECP2, and discuss therapeutic applications in CNS and liver diseases. The company recently submitted a CTA for AX-0810, targeting NTCP for cholestatic diseases.
ProQR Therapeutics (Nasdaq: PRQR) has submitted a Clinical Trial Application (CTA) to the European Medicines Agency for its first-in-human Phase 1 trial of AX-0810. This marks the first clinical development milestone for ProQR's Axiomer™ RNA editing platform.
The trial will evaluate AX-0810, an ADAR-mediated RNA editing oligonucleotide targeting NTCP, a liver cell protein. The study will assess safety, tolerability, pharmacokinetics, and target engagement in healthy volunteers, with initial data expected in Q4 2025. The treatment aims to reduce toxic bile acid accumulation in the liver for patients with cholestatic diseases.
ProQR Therapeutics (NASDAQ: PRQR) has announced its upcoming presentations at two major scientific conferences in May 2025. At the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting in New Orleans (May 13-17), the company will present one oral presentation and two poster presentations focusing on their Axiomer™ RNA editing technology. At the TIDES USA 2025 Conference in San Diego (May 19-22), they will deliver a podium presentation about advancing their Axiomer ADAR RNA editing platform.
The presentations will showcase applications including treating cholestatic diseases, addressing hepatic steatosis, and treating Rett Syndrome through RNA editing. Chief Scientific Officer Gerard Platenburg and Chief ADAR Scientist Peter A. Beal will lead the presentations, demonstrating the company's progress in developing RNA editing therapies.
ProQR Therapeutics (Nasdaq: PRQR), a company focused on RNA therapies using its Axiomer™ RNA editing technology platform, has announced its participation in the upcoming Citizens (JMP) Life Sciences Conference. The company's management will present in a fireside chat format on May 8, 2025, at 11:30 AM EDT in New York City. Interested parties can access the live webcast through ProQR's website in the "Investors & Media" section under "Events", with a replay available for approximately 30 days following the presentation.
ProQR Therapeutics (Nasdaq: PRQR), a company focused on RNA therapies using its Axiomer™ RNA editing technology platform, has announced its Annual General Meeting (AGM) of Shareholders. The meeting is scheduled for June 3, 2025, at 15:30 CEST and will be held at Allen Overy Shearman Sterling LLP's offices in Amsterdam, Netherlands.
All meeting-related documents and information will be accessible through ProQR's website under the "Investors & Media" section and on the SEC's website. Shareholders interested in attending must register according to the provided instructions in the notice and agenda.