uniQure N.V. (QURE) today announced that a Type A meeting with the FDA has been scheduled to discuss the Biologics License Application (BLA) data package intended to support accelerated approval of AMT-130, its investigational gene therapy for Huntington's disease.
Huntington's disease is a rare, inherited neurodegenerative disorder characterized by progressive motor dysfunction, cognitive decline, and psychiatric symptoms, ultimately leading to premature death. There are currently no approved disease-modifying therapies for this condition, underscoring the potential significance of uniQure's approach.
AMT-130 is uniQure's lead candidate, a one-time adeno-associated virus (AAV) gene therapy designed to deliver microRNA sequences directly into the brain to silence the huntingtin gene (HTT), which drives disease progression.
Early clinical data have suggested that the therapy can lower mutant huntingtin protein levels, potentially slowing or halting disease progression.
The FDA granted AMT-130 Breakthrough Therapy designation in April 2025, based on Phase 1/2 data compared to external controls, and Regenerative Medicines Advanced Therapy (RMAT) designation in May 2024, reflecting the therapy's potential to address a serious unmet medical need.
As of its most recent quarterly update, uniQure reported cash, cash equivalents, and investments of approximately $694.2 million. QURE has traded between $7.76 and $71.50 over the past year. The stock is currently trading in the pre-market at $26.88, up 16.11%.
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