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 progression at 24 months based on Composite Unified Huntington’s Disease Rating Scale scores compared to natural history data, which served as an external control. Read the press release here: https://uniqure.gcs-web.com/node/12041/pdf.
 
For details of all ongoing and completed EHDN-endorsed studies, click here.