UniQure has reported groundbreaking phase I/II trial results for its gene therapy AMT-130 targeting Huntington’s disease. The treatment achieved a 75% reduction in disease progression over three years, marking a major milestone for this currently untreatable neurodegenerative disorder. UniQure plans to submit for FDA approval in early 2026, potentially offering the first effective therapy to slow Huntington’s and improve patient quality of life substantially. This gene therapy utilizes microRNA to silence the mutant huntingtin gene responsible for the disease. The positive clinical data also drove a significant boost in UniQure’s stock price, affirming market confidence in the therapy’s potential impact.