Gene therapy shows signs of slowing Huntington’s disease in early trial, uniQure says

An experimental gene therapy from uniQure appears to slow progression of Huntington’s disease in a “pivotal” Phase 1/2 study, the company announced, positioning the treatment as a potential first-of-its-kind genetic therapy for the condition.

The Amsterdam-based drugmaker said that participants who received a higher dose of its therapy, AMT-130, experienced a 75% reduction in disease progression over 36 months, compared with expectations from natural history data. The treatment was “generally well-tolerated” with a “manageable” safety profile, according to the company, though full results have not yet been published in a peer-reviewed journal.

UniQure plans to submit its AMT-130 data to the US Food and Drug Administration in the first quarter of 2026, with potential US launch later that year if approved. “These findings reinforce our conviction that AMT-130 could fundamentally reshape care for Huntington’s disease and support the promise of one-time, precisely delivered gene therapies for neurological disorders,” said Dr. Walid Abi-Saab, uniQure’s chief medical officer, in Wednesday’s announcement.

Huntington’s disease is an inherited, progressive brain disorder caused by a genetic mutation, leading to the gradual breakdown of nerve cells and resulting in uncontrolled movements, behavioral changes, and cognitive decline. It is rare—affecting up to an estimated 7 in 100,000 people, most commonly of European ancestry—and currently has no cure or disease-slowing therapies, only treatments aimed at symptoms.

The Phase 1/2 trial, run with University College London, enrolled 29 adults who received either low or high doses of AMT-130 and were followed for three years. The therapy is delivered surgically via direct injection into the brain’s striatum—a region central to motor, cognitive, and emotional functions—where neurons are especially vulnerable in Huntington’s disease. The most common adverse events were related to the surgical procedure and resolved, the company said.

Investigators tracked disease progression using the Unified Huntington’s Disease Rating Scale across motor, cognitive, behavioral, and functional domains and monitored cerebrospinal fluid levels of neurofilament light protein (NfL), a marker of neurodegeneration. On average, participants saw an 8.2% decrease in NfL, which is typically elevated in more severe disease.

The Huntington’s Disease Society of America called the results a source of “immense hope” while emphasizing caution. “For the approximately 42,000 Americans and their families living with Huntington’s disease, this is a truly transformative development,” said Amy Gray, HDSA’s president and CEO, noting that there have been no therapies to date that slow the disease itself.

Independent experts also urged careful interpretation. Dr. David Rubinsztein, professor of molecular neurogenetics and deputy director of the Cambridge Institute for Medical Research, called the findings “very promising” and “exciting,” citing consistent signs of slowed progression across multiple measures. However, he noted in a statement via the UK’s Science Media Centre that the early trial included a modest number of patients and relied largely on natural history comparisons rather than a placebo control. He added that no low-dose efficacy data were reported, suggesting benefits may be limited to the higher dose—acceptable if that dose remains well-tolerated.