Gene Therapy Slows Huntington’s Progression by 75% in Three-Year Trial

Overview

• Investigators reported that high-dose recipients in a 29-patient phase I/II study had a 75% reduction in clinical progression over three years on the composite cUHDRS measure.
• Treated patients showed about 60% less loss of daily functional abilities and posted improvements on cognitive tests including Stroop and SDMT.
• Biomarker data indicated reduced neuronal injury, with cerebrospinal fluid neurofilament light (NfL) levels falling by 8.2% versus the rise expected as disease advances.
• AMT-130 was given once directly into the brain during a neurosurgical procedure lasting roughly 12 to 20 hours.
• Safety monitoring found the therapy was generally well tolerated, with adverse events mostly related to surgery and no new serious drug-related events reported since December 2022; uniQure says it plans a BLA submission in Q1 2026 under FDA expedited pathways.