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uniQure Provides Regulatory Update on AMT-130 for Huntington’s Disease
LEXINGTON, Mass. and AMSTERDAM, Dec. 04, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative
About this update from Uniqure N.v.
[{"type":"text","content":"LEXINGTON, Mass. and AMSTERDAM, Dec. 04, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the company received final meeting minutes from the U.S. Food and Drug Administration (FDA) regarding a pre-Biologics License Application (BLA) meeting held on October 29, 2025 to discuss the application for AMT-130, an investigational gene therapy for Huntington’s disease (HD). In the final meeting minutes, and consistent with uniQure’s November 3, 2025 press release, the FDA conveyed that data submitted from the Phase I/II studies of AMT-130 are currently unlikely to provide the primary evidence to support a BLA submission. uniQure is carefully evaluating the feedback and plans to urgently request a follow-up meeting with the FDA to take place in the first quarter of 2026. “We are committed to collaborating with the FDA to advance AMT-130 to patients and their families as rapidly as possible,” said Matt Kapusta, chief executive officer at uniQure. “The support we have seen these last weeks from the Huntington’s disease community, including patients, families, caregivers, clinicians and advocates reinforces the urgency of the unmet need in Huntington’s disease.” About uniQure uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. The approvals of uniQure’s gene therapy for hemophilia B – an historic achievement based on more than a decade of research and clinical development – represent a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. uniQure is now advancing a pipeline of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases. www.uniQure.com uniQure Forward-Looking Statements This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as \"anticipate,\" \"believe,\" \"could,\" “establish,” \"estimate,\" \"expect,\" \"goal,\" \"intend,\" \"look forward to\", \"may,\" \"plan,\" \"potential,\" \"predict,\" \"project,\" “seek,” \"should,\" \"will,\" \"wo...