Huntington's breakthrough: gene therapy slows progression by 75%, offering new hope but facing a long regulatory road

A small gene-therapy study for Huntington's disease reported a 75% slowing of disease progression in 29 participants over 36 months, researchers said on Wednesday. The treatment, which combines gene therapy with gene silencing to reduce levels of the toxic huntingtin protein, has not yet been published in full and remains experimental. If validated, the results would represent one of the most significant advances in a disease that currently has no approved disease-modifying therapy.

In Swindon, Gemma Botting and her husband Matt — diagnosed with Huntington's in 2011 — describe the news as life-changing. Huntington's disease typically blends features of dementia, Parkinson's and motor neurone disease and is usually fatal within about two decades of symptoms appearing. Matt began showing signs about two years ago but can still walk, though his movement is increasingly laboured and his personality has shifted; he has experienced anger outbursts and a reduction in empathy, particularly when tired. The couple, who have two children, Amelie, 11, and Hugo, eight, have long faced decisions about passing the gene to their children. They chose to conceive naturally and have embryos tested for the gene; they faced a difficult choice in a second pregnancy when the test was positive, opting to continue nonetheless. Gemma reflects on their decision: “We thought carefully about what we wanted to do. In the Huntington's community not everyone agrees with screening it out. But we both knew we did not want our children growing up with the possibility of having this gene. It is such a cruel, cruel disease.” She adds that the prospect of a treatment has altered their outlook: “Hope where there was no hope.”

The family has sought to make the most of the time they have together. When Matt was diagnosed, they paused work in the logistics industry to travel, spending a year exploring the world and taking holidays before starting a family. Gemma says their experiences have sharpened their sense of urgency about quality time together, a sentiment that has resonated with others coping with Huntington's and related disorders. She works as an evening counsellor, often supporting people who have just learned they carry the gene or are living with the disease, and she says the news of a potential therapy has given the community a lift. “Our neighbours and friends are talking about fundraising so we can pay for the treatment. I just hope it is made available on the NHS quickly,” she says. The wider Huntington's community has long faced limited options and grim prognoses; BBC listeners and viewers have contacted the outlet with stories of families who have lost multiple generations or who have faced suicide as a result of the illness.

The path to obtaining a potential therapy on the NHS remains uncertain. UniQure, the company behind the trial, has said it will apply for a license in the United States in the first quarter of 2026. If regulators in the U.S. approve the treatment for use, the UK Medicines and Healthcare products Regulatory Agency (MHRA) could leverage that work and run a faster regulatory process to assess safety and efficacy in Britain and Wales. A separate decision by the National Institute for Health and Care Excellence (NICE) would determine whether the therapy is affordable for the NHS; Scotland has its own process. Because the therapy involves brain surgery, experts say it will be expensive and restricted to specialist centers, at least initially. But NICE sources noted that high-cost gene therapies have been approved in other conditions, suggesting cost alone would not necessarily block access if value can be demonstrated. The earliest possible licensing decision for Huntington's in the UK is projected for the first half of 2027, provided all steps proceed smoothly and data withstand outside review. The current release is based on headline findings; full data and independent validation will be essential to confirm the effect size and durability.

Experts caution that the trial’s small size — 29 participants followed for 36 months — means the results require replication and longer observation to establish durable benefit. Still, the magnitude of the signal in a disease with no disease-modifying options has drawn strong reactions. Prof David Rubinsztein, deputy director of the Cambridge Institute for Medical Research, described the work as offering “real hope” for Huntington's and potentially for other neurodegenerative conditions such as Parkinson's disease, motor neurone disease and dementia, if the approach is validated in coming months. He noted that long-term benefits remain to be seen and that safety considerations will be crucial as the field advances.

What makes Huntington's disease uniquely challenging is a mutation in the huntingtin gene. If one parent carries the altered gene, a child has a 50% chance of inheriting it. The mutation leads the huntingtin protein to become toxic to neurons, driving the degenerative process that underlies the disease. The therapy aims to reduce levels of this toxic protein permanently, ideally with a single treatment. The approach merges cutting-edge gene therapy with gene-silencing technologies and marks a notable step forward in the use of genetic medicine for brain disorders.

The practical implications for patients hinge on multiple factors, including access to specialized surgical centers, regulatory approvals, and the ability to fund a potentially expensive treatment. Even if licensed, the therapy is unlikely to be a universal cure for Huntington's but could help a subset of patients in the early stages or before symptoms fully develop. Communities affected by the disease have expressed cautious optimism, balancing relief at the prospect of a therapeutic option with the realities of cost, infrastructure, and long-term effectiveness.

Huntington's disease is a hereditary neurodegenerative disorder caused by a mutation in the huntingtin gene. The condition disrupts brain function, leading to a combination of movement problems, cognitive decline and psychiatric symptoms. As with many genetic diseases, the question of how best to intervene remains complex, but researchers and patients alike are hopeful that breakthroughs in gene therapy could alter the course of the illness for some people. This potential treatment illustrates both the promise and the challenge of translating laboratory advances into widely available clinical care.

If approved and funded, the treatment would nearly certainly begin with a limited rollout at specialized centers before broader access is considered. For families like Gemma and Matt, the possibility of slowing or altering the disease’s trajectory offers a powerful new narrative — one that shifts the focus from managing decline to extending meaningful years with loved ones. As researchers continue to publish and validate the findings, patients and clinicians will watch closely for confirmation of durability, safety, and real-world impact on quality of life.

For families and clinicians, the next several months will be a critical period for scientific verification, regulatory alignment and policy planning. While the news has sparked renewed hope, the path to wider access remains contingent on robust data, cost-effectiveness analyses, and the complex logistics of delivering a brain-surgery-based therapy at scale.

As scientists and policymakers weigh the potential of this gene-based intervention, families affected by Huntington's will continue to demand clarity about when treatments might be available on the NHS and how decisions about access will be made. The journey from a headline-grabbing result to a widely accessible option is long and uncertain, but the momentum generated by this early signal has already altered the outlook for many living with Huntington's and those who care for them.