Huntington's Disease Treated Successfully For The First Time In UK Gene Therapy Trial, Researchers Report Breakthrough

(MENAFN- Live Mint) Huntington's disease , one of the most devastating inherited brain disorders , has been treated successfully for the first time. A gene therapy trial led out of the UK has shown dramatic results, slowing the disease's progress by three-quarters after three years.

The illness , which is passed down through families, is triggered by a single faulty gene. Once active, it steadily destroys brain cells, leading to mood changes, uncontrolled movements, dementia, and, in time, death. Until now, there has been no treatment that could slow it down.

“This is absolutely huge. I'm really overjoyed,” said Prof. Sarah Tabrizi, who directed the trial at University College London's Huntington's Disease Centre.“We now have a treatment for one of the world's more terrible diseases,” she told The Guardian.

How the therapy works

The treatment, known as AMT-130, is delivered through a lengthy surgical procedure lasting 12 to 20 hours. Doctors use a micro-catheter to slowly infuse a modified virus directly into two areas of the brain. Once inside, the virus carries a strand of DNA that switches off production of the toxic protein“huntingtin”, the root cause of the illness.

Patients only need one shot. But the complexity of the procedure means it will be expensive if rolled out widely, BBC reported.

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Results of the trial

The study, conducted by Dutch biotech company uniQure, followed 29 patients in the UK and the US. According to their study, three years after treatment, patients who received the high dose of the therapy had a 75% slowing of disease progression compared with the control group. Motor skills, memory, and day-to-day function all declined far less than expected.

Markers in the brain also told the same story. Levels of neurofilaments, a sign of cell death, were significantly lower in those given the drug.

“These findings reinforce our conviction that AMT-130 has the potential to fundamentally transform the treatment landscape for Huntington's disease,” Dr Walid Abi-Saab, uniQure's chief medical officer, said in the press release issued by the company.

What it means for families

Around 6,000–10,000 people live with Huntington's disease in the UK, while at least 20,000 more carry the faulty gene. Only a fraction currently tests for it, as until now, there has been no hope of slowing the disease.

“Now I think many more people will come forward for the genetic test because there's a treatment,” Tabrizi told The Guardian.

uniQure said it intends to submit the results to US regulators in early 2026. For now, researchers and patient groups describe the findings as the most convincing progress yet against a disease long seen as untreatable.

FAQsWhat is Huntington's disease?

It is a rare genetic brain disorder that causes movement, memory, and mood problems, eventually leading to dementia and death.

What did the UK trial show?

The trial found that a single-shot gene therapy slowed disease progression by about 75% after three years.

Who led the study?

The trial was led by Prof. Sarah Tabrizi, director of the University College London Huntington's Disease Centre.

What is the new treatment called?

The therapy is known as AMT-130 and was developed by the biotech company uniQure.

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