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 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.
ProQR Therapeutics (Nasdaq: PRQR) has announced key leadership appointments to support its next growth phase, focusing on its Axiomer™ RNA editing technology platform. Dennis Hom joins as Chief Financial Officer, bringing over 25 years of financial leadership and experience in raising $4.5 billion in capital and executing transactions worth $57 billion. Dr. Cristina Lopez Lopez assumes the role of Chief Medical Officer, contributing 20 years of global leadership in translational R&D.
Hom will oversee corporate development functions, including finance, investor relations, and business development. Dr. Lopez Lopez will lead preclinical, translational, clinical, and regulatory activities. Current CFO Jurriaan Dekkers and Chief Corporate Development Officer René Beukema will step down but remain during the transition period.
ProQR Therapeutics (NASDAQ: PRQR) has reported its financial and operating results for 2024, highlighting significant progress in its RNA editing pipeline. The company ended 2024 with €149.4 million in cash, providing runway into mid-2027.
Key developments include anticipated clinical data readouts for four programs in 2025-2026, led by AX-0810 targeting NTCP for Cholestatic diseases. The company successfully completed an $82.1 million public offering in October 2024 and expanded its collaboration with the Rett Syndrome Research Trust for up to $9.2 million.
Financial highlights show research and development costs increased to €36.4 million in 2024 (vs €25.1 million in 2023), while general and administrative costs decreased to €13.7 million (vs €16.2 million in 2023). Net loss for 2024 was €27.8 million (€0.32 per share), compared to €27.7 million (€0.35 per share) in 2023.
ProQR Therapeutics (NASDAQ: PRQR) announced an expanded collaboration with the Rett Syndrome Research Trust (RSRT), securing an additional $8.1 million in funding, bringing the total to $9.1 million. The funding will support the advancement of AX-2402, a therapeutic candidate based on ProQR's Axiomer RNA editing platform, into clinical trials.
The treatment targets individuals with Rett syndrome who have the R270X mutation in MECP2 gene. Rett Syndrome, affecting approximately 350,000 people worldwide, predominantly girls, is a rare neurodevelopmental disorder with no current cure. The expanded collaboration focuses on optimizing therapeutic candidates targeting the MECP2 protein and advancing them toward clinical development.
ProQR Therapeutics (Nasdaq: PRQR) has appointed Dr. Peter Beal as Chief ADAR Scientist. Dr. Beal, a professor of Chemistry at the University of California, Davis, and a former Scientific Advisory Board member, will oversee platform optimization and scientific developments in RNA editing oligonucleotides design.
As Chief ADAR Scientist, Dr. Beal will leverage his extensive expertise in ADAR biology and RNA chemistry to advance ProQR's Axiomer™ RNA editing technology platform. His research has focused on ADAR-mediated RNA-editing mechanisms, including applications for treating conditions like Rett syndrome, Alzheimer's disease, and Parkinson's disease.