Overview

• UniQure reported that in a 29-patient study, the high-dose group showed an average 75% reduction in clinical progression at 36 months on combined motor, cognitive and functional measures.
• The therapy, AMT-130, delivers genetic instructions via an adeno-associated virus during 12–20 hours of neurosurgery targeting the caudate and putamen to reduce mutant huntingtin production.
• Cerebrospinal fluid neurofilament levels, which typically rise as neurons are injured, were lower than baseline three years after treatment, suggesting reduced neuronal damage.
• Investigators described the treatment as generally well tolerated, with some patients experiencing inflammatory reactions such as headaches and confusion that resolved or were treated with steroids.
• UniQure says it plans a US regulatory submission in early 2026, with UK and European talks to follow, while experts note the need for full data, larger trials, and solutions to surgical complexity and anticipated high cost; preventive studies are being explored in gene carriers without symptoms.