Gene Therapy AMT-130 Slows Cognitive Decline in Huntington’s Disease Trial

Exam-focused facts from the 17 January 2026 current affairs briefing.

Key facts

AMT-130 gene therapy reduced cerebrospinal fluid neurofilament light levels below baseline after three years, indicating active neuroprotection.
The therapy slowed cognitive decline, particularly in processing speed and reading ability, in 29 clinically diagnosed Huntington’s disease patients aged 25–65.
Somatic expansion of the CAG repeat in Huntingtin gene accelerates disease progression and explains variable onset among individuals with identical inherited CAG length.
Early sustained attention deficits linked to inferior frontal gyrus changes appear decades before motor symptoms in HD gene expansion carriers.
HD-Young Adult Study recruited 131 participants approximately 24 years before predicted onset to detect early neurodegeneration markers and cognitive flexibility disruption.