First human gene therapy trial in Huntington’s disease


The focus of the first trial is safety and tolerability – finding out whether there any harmful or unpleasant effects of receiving AMT-130 treatment.

UniQure also includes efficacy in the stated aims of the study: that means getting an idea of whether the treatment is doing what it’s supposed to do. In the broader sense, that means slowing the progression of Huntington’s disease. It’s theoretically possible, but very unlikely, that this small first trial will show evidence of slowed progression. A more achievable aim is to test whether treatment reduces huntingtin production, which we can now measure using techniques we’ve described on HDBuzz here

The AMT-130 trial will be based at HD clinical sites in the Unites States. We don’t know what sites or how many, yet. These will be publicly announced when they come online. 

The trial will enroll just 26 patients with early symptoms of Huntington’s disease. That means people with abnormal movements, within the first few years after diagnosis was ‘officially’ confirmed by a neurologist. The age range is 25 to 65 years of age.