Huntington’s disease, a fatal genetic disorder, has just been successfully treated for the first time.

A group of scientists at the University College London and a company called uniQure facilitated the study and experiments for this breakthrough. 

UniQure created a new gene therapy, AMT-130, that the scientists at University College London have tested for the last 36 months. This therapy was used on 29 patients and was shown to slow the progression of the disease by 75%.

What is Huntington’s disease?

Huntington’s disease is a genetically inherited condition that affects about 40,000 people in the US. The disorder progressively attacks the brain over time. Symptoms include troubled walking, slurred speech, uncontrolled twitching, mood swings and trouble remembering and processing information. A person can carry the mutated huntingtin gene for their whole life, but they may not start showing these symptoms until the age of 30-40.

Because of its genetic origins, gene therapy has been the most effective treatment for Huntington’s disease. The therapy selectively replaces or repairs mutated genes with genetic material. 

AMT-130 will be the first licensed treatment that can actually slow Huntington’s disease. One dose of the new gene therapy treatment is expected to last a person’s whole life. Previously, carriers of the disease could only take medicines or therapies to help them lessen its symptoms, but this form of treatment was only marginally effective. 

It is University College London’s goal to make Huntington’s disease something that families across the world won’t have to worry about in the future — and they just might reach that goal.

This new treatment is an important scientific breakthrough for treating the incurable disease. Although it is very expensive, this development is the first step scientists have made toward a bright future for patients affected by Huntington’s disease.

UniQure hopes to get FDA approval so the treatment can be widely available by late 2026.