FDA Breakthrough Therapy Designation for AMT-130

uniQure announced on 17 April 2025 that the FDA has granted Breakthrough Therapy designation for AMT-130 gene therapy in the treatment of HD, meaning that development and review will be expedited. This designation is supported by clinical data from ongoing Phase I/II trials, including interim data announced in July 2024 showing dose-dependent slowing of disease […]

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GENERATION HD2 to continue

On 17 April 2025, Roche released a community letter announcing that GENERATION HD2 will continue following planned interim analysis by the independent Data Monitoring Committee. Enrollment has completed for this randomised, double-blind study of tominersen, but participants on the 60 mg dose will move to the higher dose (100 mg every 16 weeks), given the […]

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Clinical Trial Update: uniQure alignment with FDA on path forward for AMT-130 in HD

Clinical Trial Update: On 10th December, uniQure shared the exciting news about alignment with the US Food and Drug Administration (FDA) on a path forward for AMT-130 gene therapy in Huntington’s disease. The FDA agreed that the basis for a Biologics License Application for Accelerated Approval of AMT-130 may include: 1. Data from ongoing phase […]

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