Sep 2, 2026 —
uniQure has submitted an application to the U.S. Food and Drug Administration (FDA) for AMT-130, its investigational AAV gene therapy for Huntington’s disease, in what could become the first approved treatment targeting the underlying cause of the disease. The company has requested priority review and has also completed a submission to U.K. regulators.
Huntington’s disease is a fatal, inherited neurodegenerative disorder caused by a mutation in the huntingtin gene, and no disease-modifying therapy is currently approved. AMT-130 is a one-time, adeno-associated virus (AAV)-delivered gene therapy administered directly to the brain, designed to lower production of the disease-causing huntingtin protein.
The applications are based on previously reported three-year data indicating that AMT-130 significantly slowed signs of disease progression relative to an external natural-history comparison. uniQure is seeking accelerated approval, which would require confirmatory evidence after approval; if the FDA grants priority review, the company said a decision could come in about eight months.
If approved, AMT-130 would mark a milestone for AAV gene therapy in a large neurodegenerative indication that has long lacked disease-modifying options.
