Tag Archives: AMT-130

experimental gene therapy, AMT-130,

Overview of the Breakthrough On September 24, 2025, uniQure, a Dutch biotechnology company, announced preliminary results from a phase 1/2 clinical trial of their experimental gene therapy, AMT-130, marking the first evidence of a treatment that meaningfully slows the progression of Huntington’s disease (HD). HD is a rare, inherited neurodegenerative disorder caused by a mutation […]