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 Therapeutics (PRQR) appointed Chris Hart, PhD, as Chief Data and AI Officer and Thomas Wolf, PhD, to its Board as strategic AI advisor to advance AI-enabled RNA editing drug discovery and development.
Hart previously led Data Science and AI/ML in Genetic Medicines at Eli Lilly and earlier held leadership roles at Creyon Bio and Ionis Pharmaceuticals. He will direct ProQR’s AI strategy, linking proprietary data, models and experimental capabilities to accelerate Axiomer™ Editing Oligonucleotide (EON) design, strengthen candidate selection and inform clinical development. Wolf, co-founder and Chief Science Officer of Hugging Face, will advise the Board on technology strategy and scaling AI across the Axiomer platform.
ProQR reports that AI-enabled discovery produced AX-0811 with approximately 60% editing efficiency in humanized models, about six-fold higher than predecessor AX-0810, and reduced discovery timelines from about three years to approximately three months. The company expects up to five clinical data readouts over the next 12 months across four development programs, with target engagement data from the first two AX-0811 cohorts anticipated in early January 2027.
ProQR Therapeutics (PRQR) has dosed the first participant in its Phase 1 clinical trial of AX-0811 in healthy volunteers. AX-0811 is an AI-discovered investigational RNA editing oligonucleotide designed to modulate NTCP and is being developed as a potential disease-modifying treatment for cholestatic liver diseases, including biliary atresia. The study builds on the company’s clinical experience with the related candidate AX-0810 and aims to generate initial target engagement data for the first two cohorts in early January 2027.
ProQR will also host a virtual investor and analyst educational event, “NTCP Modulation in Biliary Atresia via Liver-Targeted RNA Editing,” on September 30, 2026 at 11 am ET. The webcast will feature ProQR management and professor Gideon Hirschfield and will review AX-0810 Phase 1 target engagement data, the role of NTCP modulation in cholestatic liver disease, and the translational rationale and development strategy for AX-0811.
ProQR Therapeutics (PRQR) will participate in two investor conferences in early September 2026 to discuss its RNA editing platform and pipeline.
Management will hold one-on-one meetings at the Wells Fargo 21st Annual Healthcare Conference in Boston on Tuesday, September 8, 2026. ProQR will also join the Cantor Global Healthcare Conference in New York on Wednesday, September 9, featuring a fireside chat at 11:30 a.m. ET and one-on-one meetings. Webcast details, where available, will be posted in the “Investors & Media > Events” section of ProQR’s website, with archived webcasts accessible for about 30 days after each presentation.
ProQR (Nasdaq: PRQR) reported second quarter 2026 results, highlighting first clinical validation of its Axiomer RNA editing platform via NTCP modulation with AX-0810 in healthy volunteers. Data showed up to an 8-fold increase in total serum bile acids at 6 mg/kg, exceeding the predefined 2-fold threshold, with favorable safety, no serious adverse events or pruritus, and an estimated eight-week half-life.
The company submitted a CTA to the EMA for AX-0811, with initial data in healthy volunteers anticipated by year-end 2026, and outlined multiple 2027 milestones, including an IIT in pediatric biliary atresia and first-in-patient data for AX-0422 and AX-2911.
Financially, ProQR closed $59.2 million in equity financing, ending Q2 2026 with €117.1 million in cash and cash equivalents, compared to €92.4 million at year-end 2025, extending cash runway into mid-2028. Q2 revenue increased to €8.8 million, with net loss narrowing to €8.7 million, or €0.08 per share.
ProQR (Nasdaq: PRQR) priced an underwritten registered direct offering of 27,624,310 ordinary shares at $1.81 per share, targeting $50 million in gross proceeds. A concurrent private placement to Eli Lilly adds about $9.2 million, both expected to close around June 26, 2026.
According to ProQR, net proceeds plus existing cash will mainly fund research and clinical development, alongside working capital and general corporate purposes.
ProQR (Nasdaq: PRQR) reported positive Phase 1 target engagement data for AX-0810, its first Axiomer RNA editing candidate, in healthy volunteers. AX-0810 showed dose-dependent NTCP modulation with up to 8-fold increases in total bile acids, favorable safety, and an ~8-week half-life, supporting advancement of next-generation NTCP candidate AX-0811 and development in biliary atresia.
ProQR (Nasdaq: PRQR) reported Q1 2026 results and a pipeline update. Cash and cash equivalents were €81.1 million, providing runway into mid-2027. Net loss was €13.4 million (€0.13 per diluted share).
Key milestones include AX-0810 Phase 1 target engagement data in Q2 2026, an IIT in pediatric biliary atresia with data in H1 2027, and CTA filings for AX-0811 in mid-2026 and AX-0422 in early 2027. The company also advanced its Axiomer RNA editing platform through AI-enabled discovery, a strategic partnership with Ginkgo Bioworks, and formation of an AI Advisory Board.
ProQR (Nasdaq: PRQR) will present multiple new data sets on its Axiomer™ RNA editing platform at major 2026 conferences, including TIDES USA, ASGCT, the RNA Society meeting, and EASL. Highlights cover liver and CNS programs, including AX-0422, AX-2402, AX-2911, and progress of lead program AX-0810 toward initial clinical data in healthy volunteers expected later this quarter.
ProQR (Nasdaq: PRQR) announced its Annual General Meeting of Shareholders will be held on June 2, 2026 at 15:00 CEST at Allen Overy Shearman Sterling LLP offices in Amsterdam. Relevant meeting documents, including the notice, agenda and explanatory notes, will be available on ProQR's investor website and on the SEC website.
Shareholders who wish to attend must register as described in the notice and agenda.
ProQR (Nasdaq: PRQR) announced the nomination of Dr. Lykke Hinsch Gylvin to its Board of Directors on April 30, 2026. Dr. Hinsch Gylvin is Chief Medical Officer and Head of Global Medicine at Boehringer Ingelheim with more than 20 years of global drug development experience.
The nomination will be submitted for shareholder approval at ProQR’s upcoming Annual General Meeting; if appointed, she will serve as an independent non-executive director.