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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 news12 items

All news

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 Announces Partnership with Ginkgo Bioworks and Formation of AI Advisory Board

ProQR and Ginkgo Bioworks to collaborate on autonomous lab high throughput data generation to support ProQR’s AI-enabled drug discovery for Axiomer™ ProQR establishes AI Advisory Board with leaders from industry and academia ProQR’s first AI-discovered Development Candidate expected to enter the clinic in 2026 LEIDEN, Netherlands & CAMBRIDGE, Mass., April...

ProQR to Host Virtual Investor and Analyst Event Highlighting Pipeline on April 8, 2026

LEIDEN, Netherlands & CAMBRIDGE, Mass., March 25, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today announced that it will host a virtual Investor and Analyst Event highlighting its...

ProQR Announces Year End 2025 Operating and Financial Results

Advancing AX-0810 through ongoing Phase 1 dosing, with target engagement data expected 1H 2026 Selected Development Candidates AX-2402 for Rett syndrome (MECP2, R270X) and AX-2911 for MASH (PNPLA3) Ended 2025 with € 92.4 million cash and cash equivalents and achieved $4.5 million in Eli Lilly collaboration milestones in 2025, supporting runway into mid-2027...

ProQR Announces Webcast of Presentation at the 2026 Citizens Life Sciences Conference

LEIDEN, Netherlands & CAMBRIDGE, Mass., March 03, 2026 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (ProQR), a 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 present at the Citizens Life...

ProQR Announces Planned Changes to Board Composition

LEIDEN, Netherlands & CAMBRIDGE, Mass., Feb. 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 Axiomer™ RNA editing technology platform, today announced that Dinko Valerio, a co-founder of the Company, and Alison...

ProQR Therapeutics N.V. • Endpoint Arena