For families affected by Huntington’s disease, hope often feels like a distant promise. But on September 24, 2025, a wave of cautious optimism swept through the medical community and among patients, as researchers in the United Kingdom announced a breakthrough: for the first time, the progression of Huntington’s disease has been dramatically slowed—by an astonishing 75%—thanks to a pioneering gene therapy. The development, led by a team at University College London and the biotechnology company uniQure, has been hailed as a turning point in the fight against one of the most devastating hereditary brain disorders.
Huntington’s disease is a rare, inherited condition that relentlessly destroys nerve cells in the brain, leading to a combination of symptoms resembling dementia, Parkinson’s disease, and motor neuron disorders. The first signs usually appear when patients are in their 30s or 40s, and the disease is typically fatal within two decades. Until now, treatments have only managed symptoms, offering no reprieve from the inexorable decline.
But as reported by BBC News and UNN, a phase 1/2 clinical trial involving 29 patients has upended expectations. The therapy, known as AMT-130, targets the very root of the disease: the mutated huntingtin gene. This gene, when altered, produces a toxic protein that kills neurons, setting off the cascade of symptoms that devastate families for generations. If one parent carries the mutation, each child faces a 50% risk of inheriting the faulty gene.
So how does this new therapy work? It’s a marvel of modern medicine, combining advances in gene therapy and gene silencing technology. Patients undergo a 12 to 18-hour neurosurgical procedure in which a harmless virus, modified to carry a specially designed DNA sequence, is infused deep into the brain. Real-time MRI guides surgeons as they deliver the therapy to two key regions—the caudate nucleus and the putamen—both of which are heavily impacted by Huntington’s.
Once inside the brain, the virus acts as a “microscopic postman,” as described by BBC News, delivering the new genetic material into neurons. There, the cells are reprogrammed to produce microRNA, which intercepts and disables the messenger RNA instructions for making the mutant huntingtin protein. The result? Levels of the toxic protein drop, and neurons are shielded from further damage.
The trial’s results, released by uniQure and detailed by Fierce Biotech, are nothing short of remarkable. Three years after receiving the high-dose therapy, patients showed a 75% slowdown in disease progression compared to historical controls. On the Unified Huntington’s Disease Rating Scale, those treated had a mean reduction of just 0.38, compared to 1.52 in untreated individuals. In terms of daily life, patients maintained greater independence: their Total Functional Capacity scores dropped by only 0.36, versus a 0.88 drop in controls—translating to a 60% reduction in loss of functional independence.
For families, these numbers mean more than statistics. They represent years of life reclaimed. Professor Sarah Tabrizi, director of the UCL Huntington’s Disease Centre and a leader of the study, told BBC News, “We never in our wildest dreams would have expected a 75% slowing of clinical progression.” She described the results as “spectacular,” and added, “It means the decline you would normally expect in one year would take four years after treatment, giving patients decades of good quality life.”
Other markers of success were equally encouraging. Instead of the expected one-third increase in neurofilament light protein—a biomarker signaling ongoing brain cell death—patients actually saw an 8.2% reduction in their cerebrospinal fluid. This suggests the therapy isn’t just slowing symptoms, but is actively protecting neurons from degeneration.
The impact on patients’ daily lives is already visible. While none of the trial participants have been publicly identified, one who had retired due to deteriorating health has now returned to work. Others, who were expected to need wheelchairs by now, are still walking. For many, these are not just medical milestones but deeply personal victories.
Jack May-Davis, a 30-year-old barrister’s clerk who carries the Huntington’s gene, shared with BBC News how the breakthrough has transformed his outlook. “It’s absolutely incredible,” he said. “It does allow me to think my life could be that much longer.” For Jack, who watched his father succumb to the disease at 54, the news offers a future that, until now, seemed out of reach.
Of course, the road ahead is not without challenges. The therapy is expected to be expensive and complex—requiring hours of brain surgery and highly specialized care. As Professor Ed Wild, another principal investigator, noted, “It will be expensive for sure.” There’s no official price yet, but gene therapies are often among the most costly treatments available. Still, health systems like the UK’s NHS have shown willingness to fund such breakthroughs, as seen with a £2.6 million-per-patient gene therapy for haemophilia B.
Safety is another consideration. The trial was temporarily paused in 2022 after a few patients experienced side effects, including headaches and confusion due to inflammation from the viral vector. These issues resolved with or without steroid treatment, and the trial resumed. Researchers say the therapy appears safe overall, and because brain cells aren’t replaced like blood or skin cells, the effects should last a lifetime.
The commercial and regulatory implications are substantial. Following the positive results, uniQure’s share price tripled on September 24, 2025, reflecting the market’s enthusiasm. The company plans to submit for US regulatory approval in early 2026, with a launch anticipated later that year. Discussions with UK and European authorities are expected to follow.
For now, the therapy will be available only to a limited number of patients—those who can undergo the demanding surgery and meet strict clinical criteria. But researchers are already looking further ahead. Professor Tabrizi is working with young people who know they carry the gene but have not yet developed symptoms, with hopes of launching “prevention trials” to see if the disease can be delayed or even stopped entirely.
Dr. Walid Abi-Saab, uniQure’s Chief Medical Officer, summed up the sentiment: “We are incredibly excited 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 families who have lived under the shadow of Huntington’s for generations, the news marks the dawn of a new era—one where hope is no longer just a distant promise, but a tangible possibility.
