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 (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.
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.