In a monumental medical breakthrough, scientists have for the first time successfully treated Huntington’s disease, a fatal and inherited neurodegenerative disorder. The groundbreaking treatment was announced in late September 2025, following a global clinical trial led by researchers at University College London (UCL) and gene therapy company uniQure. The new gene therapy, known as AMT-130, slowed the progression of the disease by an unprecedented 75%, marking a transformative moment for patients and families affected by this devastating condition.

Huntington’s disease is caused by a mutation in the HTT gene, discovered in 1993, which produces a toxic protein that gradually destroys brain cells essential for movement, thinking, and behavior. Symptoms typically emerge in middle age, around the 30s or 40s, including involuntary movements, cognitive decline, and psychiatric problems. Without effective treatment, the disease leads to severe disability and death within approximately two decades.

The AMT-130 therapy involves a sophisticated gene-silencing technique administered via delicate brain surgery that can last between 12 to 18 hours. This invasive neurosurgical procedure introduces a viral vector designed to deliver genetic instructions that reduce the production of the mutant huntingtin protein directly in the brain. The trial’s principal investigator, Professor Edward Wild of UCL, described the results as “world-changing,” noting that some patients included in the study had drastically slowed disease progression and one previously medically retired participant even returned to work.

“This is the first time a treatment has shown statistically significant slowing of Huntington’s disease progression,” said Professor Sarah Tabrizi, director of the Huntington’s Disease Centre at UCL. “We never dreamed we would see a 75% slowing of clinical progression.” She emphasized the importance of this therapy in potentially preserving daily function and extending patients’ quality of life by years.

The trial data revealed a substantial reduction in key neurodegenerative biomarkers in cerebrospinal fluid, reinforcing the therapy’s disease-modifying effect. AMT-130 has also been generally well-tolerated with manageable safety risks, an important factor considering the complexity of the brain surgery involved.

This medical milestone brings hope to approximately 8,000 people living with Huntington’s disease in the UK alone, and many more worldwide. UniQure plans to submit applications for accelerated approval to the US FDA in early 2026, with regulatory submissions pending for the UK and European Union. The FDA has already granted the therapy Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations, facilitating a potentially faster approval process.

Families affected by Huntington’s describe the news as life-changing. The therapy is not a cure but represents the first treatment to significantly slow down a disease long considered untreatable. Experts urge continued research to improve therapies and expand access so patients globally can benefit. Future efforts will also focus on diagnosing and treating people earlier to prevent or delay symptom onset altogether.

In summary, the successful trial of AMT-130 is a watershed moment in neuroscience and genetic medicine, offering unprecedented hope for those impacted by Huntington’s disease. The next steps involve regulatory approvals, scaling treatment availability, and ongoing research to build on this success to ultimately transform Huntington’s from a fatal diagnosis into a manageable condition.