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. 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 management will present at the Citizens Life Sciences Conference on March 11, 2026 at 3:25pm ET. A live webcast and recording will be available via the company’s Investors & Media website events page. Archived webcasts will remain available for about 30 days.
ProQR (Nasdaq: PRQR) announced planned changes to its Board of Directors effective at its 2026 Annual General Meeting. Co-founder Dinko Valerio and director Alison Lawton will rotate off the Board as their terms conclude. An executive search firm has been engaged to identify new director candidates to support long-term value creation.
The Board says the changes reflect governance, succession planning, and support for advancing ProQR's clinical programs.
ProQR (NASDAQ: PRQR) reported encouraging early Phase 1 results for AX-0810: no serious adverse events and pharmacokinetics in Cohort 1 (3 mg/kg) consistent with non-clinical data after four weeks of dosing. The single-center, randomized, double-blind multiple-ascending-dose study plans up to 33 participants (24 active, 9 placebo) with target engagement data expected in H1 2026 and a patient cohort planned thereafter. ProQR also selected development candidates for AX-2402 (Rett syndrome, MECP2 R270X) and AX-2911 (MASH, PNPLA3 I148M) and reported $4.5M in 2025 collaboration milestones with Eli Lilly, supporting runway into mid-2027.
ProQR (Nasdaq: PRQR) will participate in a fireside chat at the 8th Annual Evercore Healthcare Conference on Thursday, December 4, 2025 at 8:20 AM ET. The presentation is part of the company’s investor outreach about its Axiomer RNA editing platform. Webcast access and live details are available from the company’s Investors & Media website section at www.proqr.com under "Events". Archived webcasts will be available for approximately 30 days after the presentation.
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.