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FDA approval
UniQure receives RMAT designation for AMT-130 gene therapy for Huntington’s disease
Cell & Gene Therapy
Jul 9, 2024
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Cell & Gene Therapy

Cell & Gene Therapy

Jul 9, 2024

UniQure receives RMAT designation for AMT-130 gene therapy for Huntington’s disease

FDA approval

  • UniQure has announced that the FDA has granted it the Regenerative Medicine Advanced Therapy (RMAT) designation for its developmental gene therapy for Huntington's disease.

  • The gene therapy, AMT-130, was tested in a Phase I/II trial with 21 patients for 24 months. The disease progression was measured using the composite Unified Huntington’s Disease Rating Scale. The company claims that in higher doses, AMT-130 slowed disease progression by 80%. The RMAT designation has been granted for the therapy following the reported positive results from the trials. 

  • Based in the Netherlands, uniQure is a gene therapy company specializing in developing and commercializing adeno-associated virus (AAV) based therapies. The company aims to provide novel treatments for patients with severe genetic diseases. Its leading product, AMT-060, is a gene therapy for treating moderately severe hemophilia B. The company's pipeline includes AMT-130 for Huntington's disease and AMT-150 for Spinocerebellar Ataxia type 3. UniQure leverages its proprietary gene technology platform to develop, manufacture, and test these therapies, targeting liver-directed and central nervous system (CNS) disorders​.

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