uniQure Announces FDA Breakthrough Therapy Designation Granted to AMT-130 for the Treatment of Huntington’s Disease

Core Points - The U.S. FDA has granted Breakthrough Therapy designation to AMT-130 for treating Huntington's disease, highlighting the urgent need for effective treatments in this area [2][3] - AMT-130 has also received multiple designations from the FDA, including Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Fast Track designations, indicating its potential significance in addressing unmet medical needs [2] - Interim data from ongoing Phase I/II trials show a dose-dependent slowing of disease progression in treated patients, with 45 patients having received AMT-130 to date [3] Company Overview - uniQure is a gene therapy company focused on developing transformative therapies for severe medical conditions, including Huntington's disease, ALS, and Fabry disease [6] - The company has previously achieved a significant milestone with its gene therapy for hemophilia B, marking a major advancement in genomic medicine [6] Huntington's Disease Context - Huntington's disease is a rare, inherited neurodegenerative disorder affecting approximately 70,000 diagnosed individuals in the U.S. and Europe, with no approved therapies currently available to slow its progression [5]