A revolutionary advance in gene therapy has sparked hope for Huntington’s disease patients across the UK. Scientists at University College London have revealed that a new treatment, AMT-130, has slowed the disease’s progression by 75%. This breakthrough could possibly redefine how neurodegenerative disorders are treated.
Huntington’s disease is a genetic condition that damages the brain cells over time. It causes movement issues, memory loss, and emotional instability. The disease is inherited, and each child of an affected parent has a 50% chance of developing it. Until now, treatments have only managed symptoms without changing the disease’s course.
The new gene therapy uses a harmless virus to deliver DNA into brain cells. This DNA helps produce microRNA molecules that silence the faulty gene. As a result, the toxic protein responsible for brain damage is reduced. The therapy is given during brain surgery and requires only one dose.
In a clinical trial, 29 patients received AMT-130. Seventeen received a high dose, and twelve received a low dose. Over three years, the high-dose group showed 75% less disease progression than untreated patients. Researchers used standard scales to measure motor, cognitive, and functional abilities.
Dr. Ed Wild, a neurologist at UCL, called the results “extraordinary.” He said some patients who were expected to lose mobility are still walking. One participant even returned to work after being medically retired. These outcomes suggest the therapy could improve both lifespan and quality of life.
Professor Sarah Tabrizi, who co-led the trial, expressed strong optimism. She said the results exceeded expectations and could change the future of Huntington’s treatment. Families affected by the disease now have a reason to hope.
The trial’s sponsor, uniQure, plans to submit a Biologics License Application to the US FDA in early 2026. Approval in the UK and Europe is expected soon after. The therapy has already received Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations.
Although the results are preliminary, they have excited the medical community. Experts say more testing is needed to confirm long-term safety and effectiveness. Still, the early data suggest that gene therapy could become a key tool in treating brain disorders.
This breakthrough may also help fight other diseases. Researchers believe similar gene therapy methods could treat Parkinson’s and Alzheimer’s. By targeting the genetic causes, scientists hope to develop treatments that go beyond symptom control.
In the UK, around 8,000 people live with Huntington’s disease. Many more are at risk due to its hereditary nature. For these families, the new therapy offers a future that once seemed impossible. The delivery method, though complex, is manageable. Surgeons use MRI guidance to inject the therapy into specific brain areas. The procedure takes 12 to 18 hours but requires only one dose. Most side effects were related to surgery and resolved quickly.
The treatment’s delivery method, though complex, is manageable. Surgeons use MRI guidance to inject the therapy into specific brain regions. The procedure takes 12 to 18 hours but requires only one dose. Most side effects reported were related to the surgery and resolved without complications.
As the scientific community awaits peer-reviewed publication, patients and families are already celebrating. The chance to slow or stop the disease’s progression is a major shift. For decades, Huntington’s was a diagnosis with no escape. Now, thanks to gene therapy, that story may be changing.
This achievement is not just scientific. It reflects the courage of patients who joined the trial. Their bravery has opened the door to a future where Huntington’s disease is no longer a life sentence. With continued research, the promise of gene therapy could soon become reality for thousands more.
