Gene therapy shows 75% slowing of Huntington’s disease progression in early trial

A small, early-stage trial of uniQure’s gene therapy AMT-130 found that a high dose slowed Huntington’s disease progression by about 75% over three years in 12 of 29 participants followed for three years. The Phase I/II study compared treated patients with a similar group of individuals who did not receive the therapy, and the company said the results are groundbreaking for a disease that currently has no cure. The data have not yet been published in a peer-reviewed journal, but uniQure said they will be presented at the HD Clinical Research Congress next month in Nashville.

The trial enrolled 29 participants with Huntington’s disease, a hereditary, progressive disorder that damages brain cells. In the high-dose cohort, 12 patients were tracked for three years, and the observed slowing of disease progression contrasted with the course observed in untreated patients, according to uniQure.

The therapy uses a harmless virus to deliver instructions for adding permanent DNA to brain cells, reducing the production of the huntingtin protein, which is mutated in Huntington’s disease. UniQure said a single dose is expected to provide lasting benefit, effectively a one-time, precision-delivered intervention. The company described AMT-130 as “generally well-tolerated” with a manageable safety profile at both high and low doses; most common side effects were related to the administration procedure and resolved.

Huntington’s disease is caused by a mutation in the huntingtin (HTT) gene on chromosome 4. People who inherit the mutated gene have a 50% chance of developing the condition. The mutation leads the huntingtin protein to become abnormally long and to form toxic clumps inside brain cells, disrupting function. Symptoms—such as involuntary movements, cognitive decline and mood changes—often appear in the late 30s or 40s. As nerve cells deteriorate, patients may develop dementia, speech and swallowing difficulties, and eventual immobility, necessitating round-the-clock care.

The findings come as the HD field awaits fuller, peer-reviewed publication. Dr. Walid Abi-Saab, uniQure’s chief medical officer, said, “These findings reinforce our conviction that AMT-130 has the potential to fundamentally transform the treatment landscape for Huntington’s disease, while also providing important evidence supporting one-time, precision-delivered gene therapies for the treatment of neurological disorders.”

In the context of the broader landscape, the Huntington’s Disease Society of America estimates about 42,000 Americans live with Huntington’s disease, with roughly 200,000 at risk of inheriting the condition. The disease is rooted in a mutation of the HTT gene, and there is currently no cure or treatment proven to halt or reverse its progression. Researchers emphasize that symptom management through medications, physical, speech and occupational therapy, and nutritional support remains essential.

The HD Clinical Research Congress in Nashville is slated to host presentations of the full results next month, and uniQure plans to submit its data to the U.S. Food and Drug Administration in the first quarter of 2026, with hopes of launching AMT-130 in the United States later in 2026. Siddharthan Chandran, director of the UK Dementia Research Institute who was not involved in the study, cautioned that full peer-reviewed results are still needed, but also called the development promising and noted that the next step is larger, late-stage trials. “We’ll need to wait for the full peer-reviewed results to come out, but this is promising and gives real hope to families dealing with this disease. The next step is getting this drug into larger, late-stage trials,” Chandran said.

Scientists and patient advocates alike are cautious about extrapolating early findings to broader use, but the potential for a one-time gene therapy to slow progression offers a new line of hope in Huntington’s disease research. If subsequent trials confirm the benefit and safety profile, AMT-130 could become a landmark in neurology, marking a shift toward disease-modifying, rather than solely symptomatic, interventions for Huntington’s.