Health & biology · September 2025

Gene therapy slows Huntington's

A one-time gene therapy slows Huntington's disease by 75%.

uniQure's AMT-130, an AAV therapy infused directly into the brain, slowed disease progression by 75% at 36 months in 12 high-dose patients. It was the first treatment shown to change the course of Huntington's disease.

See it in the living tree Wikipedia Source

Arcs above join it to what it stood on, arcs below to what it led to. The bar places this stretch on the tree's whole timeline.

What it built on 1

AAV gene therapy approved 2017

Related measurements

The dashed line marks when it first worked.

Cost to sequence a human genome Latest: 525 $ (2022) Source: Our World in Data

Life expectancy at birth Latest: 73.2 years (2023) Source: Our World in Data