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  • News bot April 30, 2026, 11:08 a.m.

    📋 UNIQURE N.V. (QURE) - Clinical Trial Update

    Filing Date: 2026-04-30

    Accepted: 2026-04-30 07:07:07

    Event Type: Clinical Trial Update

    Event Details:

    UNIQURE N.V. (QURE) Announces Clinical Trial Update UNIQURE N.V. (QURE) provided an update on its clinical development programs. Clinical Development Highlights:
    • Drug Program: granted, submission
    • Diseases/Conditions: AMT-130 in Huntington’s disease
    • Clinical Stage: Phase III
    • Collaboration: U.S. FDA
      • anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, among others: risks related to the Company’s Phase I/ll clinical trials of AMT-130, including the risk that such trials will be unable to demonstrate data sufficient to support further clinical development or regulatory approval; the risk that more patient data become available that results in a different interpretation then the one derived from topline data; risks related to the Company’s interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to regulatory approval; the risk that the Company will be unable to align with the FDA, MHRA, or other regulatory authorities on an approval pathway for its gene therapy candidates, including AMT-130; whether the measurements that the Company is evaluating are viewed as robust and sensitive measurements of disease progression; whether RMAT designation, Breakthrough Therapy designation, or any accelerated pathway, if granted, will lead to regulatory approval; the Company’s ability to conduct and fund a Phase III or confirmatory study for AMT-130; the Company’s ability to continue to build and maintain the infrastructure and personnel needed to achieve its goals; the Company’s effectiveness in managing current and future clinical trials and regulatory processes; the Company’s ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the continued development and acceptance of gene therapies; the Company’s ability to obtain, maintain and protect its intellectual property; and the Company’s ability to fund its operations and to raise additional capital as needed and on acceptable terms. These risks and uncertainties are more fully described under the heading "Risk Factors" in the Company’s periodic filings with the U.S. Securities & Exchange Commission (SEC), including its Annual Reports on Form 10-K, its Quarterly Reports on Form 10-Q and in other filings that the Company makes with the SEC from time to time. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements and, except as required by law, the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future.​uniQure Contacts:​​​FOR INVESTORS:FOR MEDIA:​​​​Chiara RussoTom MaloneDirect: 781-491-4371
      • expected in the third quarter of 2026

    🔬 Clinical Development Pipeline (UNIQURE N.V.):

    Product Type Development Stage Therapeutic Area Source
    rAAV2/5-hNAGLU DRUG Phase PHASE1 Sanfilippo Syndrome B ClinicalTrials.gov
    Mycophenolate mofetil DRUG Phase PHASE2 LPL Deficiency ClinicalTrials.gov
    Cyclosporins DRUG Phase PHASE2 LPL Deficiency ClinicalTrials.gov
    Prednisolone DRUG Phase PHASE2 LPL Deficiency ClinicalTrials.gov
    alipogene tiparvovec DRUG Phase PHASE2 LPL Deficiency ClinicalTrials.gov
    rAAV2/5-PBGD vector dosage 4 GENETIC Phase PHASE1 Acute Intermittent Porphyria ClinicalTrials.gov
    rAAV2/5-PBGD vector dosage 3 GENETIC Phase PHASE1 Acute Intermittent Porphyria ClinicalTrials.gov
    rAAV2/5-PBGD vector dosage 2 GENETIC Phase PHASE1 Acute Intermittent Porphyria ClinicalTrials.gov
    rAAV2/5-PBGD vector dosage 1 GENETIC Phase PHASE1 Acute Intermittent Porphyria ClinicalTrials.gov

    💼 Business Developments:

    • Partnership: Not available
    • Acquisition: Not available
    • Licensing: Not available
    • Regulatory Approval
    • Executive Changes: Not available

    Structured Data:

    • Company Name: UNIQURE N.V.
    • Ticker Symbol: QURE