Breakthrough therapy slows Huntington’s disease progression by 75%
Phase II trials with 24 patients bring hope, but experts are watching to see if results will be repeated in larger groups.
The first treatment for Huntington’s disease could be approved in 2026, following a phase II study showing a 75% reduction in the rate of disease progression.
The treatment’s developer, uniQure, is aiming for US approval late next year for the microRNA therapy, called AMT-130, which targets the huntingtin gene responsible for the condition when mutated.
The therapy is applied to several parts of the striatum in the brain’s basal ganglia using microscopic catheters during a one-time, 8-10-hour surgery.
uniQure’s phase I/II trials involved 24 patients, divided into high-dose and low-dose groups, with 36 months of follow-up.
