
By Edward Ryan
A team of researchers has successfully treated Huntington’s Disease for the first time.
The news has been described as “a game changer” by the Huntington’s Disease Association of Ireland.
Huntington’s is a degenerative disease that kills nerve cells in the brain and has been described as a combination of dementia, motor neuron disease, and Parkinson’s.
It is a very rare condition that affects thousands of people worldwide.
But on September 24th, biomedical company uniQure announced that it had successfully treated Huntington’s in a major clinical trial.
Huntington’s explained
Huntington’s is caused by a change in genes and is hereditary, passing from a parent to their children.
It is caused by a mutation in the HTT gene. This gene makes a protein called huntingin, which helps with the function of the nerves.
Huntington’s Disease restricts the DNA’s ability to produce huntingin, which causes the proteins to grow abnormally and destroy the nerves rather than help them.
This decay occurs in the area of the brain that affects movement and can also target the brain cortex, causing cognitive and behavioural issues.
Huntington’s is an inherited disease. If one of your parents has Huntington’s, then the likelihood of you developing the disease is around 50%.
Symptoms of the disease often begin to appear in people in their late thirties, with the average lifespan after first symptoms ranging from 10-30 years.
The treatment
The trial involved a gene therapy called AMT-130 which was administered to patients during brain surgery.
AMT-130 contained a virus carrying a micro-RNA that was designed to target the huntingin gene.
It was administered as a one-off during complex brain surgery that lasted anywhere from 12 to 18 hours.
The results of the study were astounding.
It showed a 75% slowing of disease progression in patients who received a high dose of the treatment after 36 months.
The study also found that there were no new drug-related serious adverse effects observed between December 2022 and the end of June 2025.
This major breakthrough will bring relief to people who have the gene for Huntington’s and there is hope that early treatment may prevent the emergence of symptoms completely.
In a statement announcing the news on Wednesday, Walid Abi-Saab, M.D., the Chief Medical Officer at uniQure said, “We are incredibly excited to about these topline results and what they may represent for individuals and families affected by Huntington’s disease.
“These findings reinforce our conviction that AMT-130 has the potential to fundamentally transform the treatment landscape for Huntington’s disease, while also providing important evidence supporting one-time, precision delivered gene therapies for the treatment of neurological disorders.”
“It’s a game changer”
Patricia Towey is the Service Manager with the Huntington’s Disease Association of Ireland . She was delighted with this announcement.
“It’s really exciting news for so many families that are really struggling with this devastating neurodegenerative disease, you know, so this news is very exciting.”
She added, “It’s the first time that the course of the disease has stopped by up to 75% for the people that have been treated. It’s three years of data and, yeah, it’s really a game changer in so many ways.”
Who is uniQure?
uniQure is a gene therapy company with teams of researchers based across the Netherlands, the UK, and the US.
They have an extensive track record in gene therapy and developed the first approved human gene therapy in the western world.
They also have worked on treatments for Haemophilia B throughout the years.
The study on AMT-130 has taken many years, but there is still a ways to go before the treatment is available to the public.
uniQure is aiming to have the treatment available in the US some time in 2026, but costs for the treatment are expected to be very high.
Meanwhile, shares in uniQure jumped over 200% across various markets in the hours following the announcement.


















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