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ProQR Therapeutics N.V.

Pipeline6 assets

  • AX-0810AX-0810 / NTCP (SLC10A1) / RNA Editing Oligonucleotide (Axiomer EON) / Liver
    Biliary AtresiaPrimary Sclerosing Cholangitis
  • AX-0811AX-0811 / NTCP / RNA Editing Oligonucleotide (Axiomer EON) / Liver
    Cholestatic Diseases
  • AX-0422AX-0422 / IDUA / RNA Editing Oligonucleotide (Axiomer EON) / Metabolic / Rare Disease
    Hurler Syndrome
  • AX-2402AX-2402 / MECP2 (R270X) / RNA Editing Oligonucleotide (Axiomer EON) / CNS / Rare Disease
    Rett Syndrome
  • AX-2911AX-2911 / PNPLA3 / RNA Editing Oligonucleotide (Axiomer EON) / Liver / Metabolic
    MASH
  • AX-1412AX-1412 / B4GALT1 / RNA Editing Oligonucleotide (Axiomer EON) / Cardiovascular
    Cardiovascular Disease

Latest news10 items

All news

ProQR Announces Appointment of Chris Hart as Chief Data and AI Officer and Thomas Wolf to join the Board of Directors as Strategic AI Advisor

Appointments support ProQR’s strategy to scale AI across the discovery and development of medicines using its Axiomer RNA editing platform and pipeline AX-0811, the first ProQR program identified through its AI-enabled discovered engine in clinical testing, with target engagement data expected in early January 2027 ProQR’s expanded AI strategy is directly...

ProQR Announces First Participant Dosed in Phase 1 Study of AX-0811 and Virtual Investor and Analyst Educational Event on September 30, 2026

LEIDEN, Netherlands & CAMBRIDGE, Mass., Sept. 09, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a clinical-stage company dedicated to changing lives through transformative RNA therapies based on its proprietary ADAR-mediated Axiomer™ RNA editing technology platform, today announced that the first participant has been dosed in the...

Trial Start • Phase 1Trial initiation Wed, Sep 9, 2026

ProQR to Participate in Upcoming Investor Conferences in September 2026

LEIDEN, Netherlands & CAMBRIDGE, Mass., Sept. 02, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a clinical-stage company dedicated to changing lives through transformative RNA therapies based on its proprietary ADAR-mediated Axiomer™ RNA editing technology platform, today announced that company management will participate in the...

ProQR Announces Second Quarter 2026 Operating and Financial Results

First clinical validation of Axiomer™ RNA editing established through NTCP modulation with positive AX-0810 clinical target engagement data; full Phase 1 dataset to be presented at a medical or scientific conference later this year Multiple clinical milestones, including initial AX-0811 Phase 1 data expected by year-end 2026, followed in H1 2027 by:...

Trial Results • Phase 1Results event Wed, Dec 31, 2025

ProQR Announces Positive Phase 1 Target Engagement Data for AX-0810, Establishing First Clinical Validation of the Axiomer RNA Editing Platform

Data from ongoing Phase 1 study of AX-0810 in healthy volunteers demonstrated dose-dependent target engagement on all key biomarkers in the evaluable 3 mg/kg and 6 mg/kg cohorts AX-0810 demonstrated up to 8-fold change (6 mg/kg) in total bile acids in serum, exceeding the 2-fold threshold identified by the Company as a meaningful indicator of NTCP...

Interim Data • Phase 1Interim data Thu, Jun 25, 2026

ProQR Announces First Quarter 2026 Operating and Financial Results

AX-0810 target engagement data in healthy volunteers on track for Q2 2026; biliary atresia selected as initial Phase 2 indication Pipeline expansion with advancement of AX-0811 (NTCP) and AX-0422 (IDUA) toward the clinic, supporting multiple upcoming clinical catalysts Continued advancement of Axiomer platform, including AI-enabled discovery capabilities,...

Interim Data • Phase 1AX-0810 target engagement data Tue, Jun 30, 2026

ProQR Announces Multiple Upcoming Scientific Conference Presentations Highlighting the Breadth of its Axiomer™ RNA Editing Platform

LEIDEN, Netherlands & CAMBRIDGE, Mass., May 11, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a clinical-stage company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced presentations at several upcoming scientific conferences,...

ProQR Announces Annual General Meeting of Shareholders to be Held June 2, 2026

LEIDEN, Netherlands & CAMBRIDGE, Mass., May 04, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a clinical-stage company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced the Annual General Meeting (AGM) of Shareholders will take place...

ProQR Announces Nomination of Dr. Lykke Hinsch Gylvin to its Board of Directors

LEIDEN, Netherlands & CAMBRIDGE, Mass., April 30, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a clinical-stage company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced the nomination of Lykke Hinsch Gylvin, MD, EMBA, Chief Medical...

ProQR Therapeutics N.V. • Endpoint Arena