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) outlined pipeline progress at its April 8, 2026 investor event, highlighting lead RNA editing program AX-0810, selection of biliary atresia for Phase 2, and multiple programs advancing toward clinic.
The company said AX-0810 target engagement data are on track for this quarter, AX-0811 CTA is expected mid-2026, and several clinical readouts are expected within a cash runway into mid-2027.
ProQR (Nasdaq: PRQR) announced an AI strategy for its Axiomer RNA editing platform, partnering with Ginkgo Bioworks and forming an AI Advisory Board. ProQR gains access to Ginkgo’s 50+ instrument autonomous lab, expects a CTA mid-2026 for its first AI-discovered candidate, and initial clinical data by year-end 2026.
The Advisory Board includes leaders from AI and techbio to guide discovery model development and best practices.
ProQR (Nasdaq: PRQR) will host a virtual Investor and Analyst Event on April 8, 2026 from 10:00 am ET covering its Axiomer™ RNA editing pipeline.
The company will highlight programs beyond AX-0810, announce new programs with upcoming clinical readouts, and unveil AX-0810's primary indication ahead of target engagement data expected in H1 2026.
ProQR (Nasdaq: PRQR) reported year-end 2025 results and a pipeline update. Key points: €92.4M cash at Dec 31, 2025, €52.8M net cash used in operations, €44.7M R&D spend, and a €42.2M net loss. Clinical progress includes AX-0810 Phase 1 dosing with target engagement data expected 1H 2026; Development Candidates selected for AX-2402 and AX-2911. The company achieved $4.5M in Eli Lilly collaboration milestones, supporting runway into mid-2027.
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.