UniQure treatment said to continue to slow Huntington’s progression

By the Editorial Team · Wednesday, September 30, 2026

UniQure’s experimental gene therapy AMT-130 demonstrated a sustained but reduced slowing of Huntington’s disease progression four years after a single brain infusion, with treated patients showing less decline on clinical scales compared to controls. While the therapy’s effect diminished since the three-year assessment, it still marked a significant advance in slowing functional decline and impacting neuronal damage markers, with regulatory review underway in the U.S. and UK.

An experimental gene therapy developed by UniQure showed continued, though diminished, slowing of Huntington’s disease progression four years after treatment, the biotech company announced this week. The therapy, called AMT-130, was surgically delivered directly into patients’ brains in a one-time procedure intended to reduce production of the mutant huntingtin protein responsible for the neurodegenerative disorder.

In the latest analysis of the ongoing study, 12 patients receiving a high dose of AMT-130 experienced a 44 percent reduction in disease progression compared to a matched external control group drawn from a natural history study. This assessment was based on cUHDRS, a standard composite scale measuring motor, cognitive, and functional symptoms of Huntington’s disease. Patients treated with AMT-130 lost an average of 0.90 points on the scale after four years, while untreated participants declined by 1.61 points over the same period. However, this difference did not reach statistical significance.

UniQure had previously reported a 75 percent slowing of disease progression at the three-year mark, and the current findings indicate some attenuation of the therapy’s effect over the additional year. Despite this, other measures from the study suggested a 61 percent slowing based on functional capacity assessments and stabilization of neurofilament light chain (NfL), a biomarker of neuronal damage found in spinal fluid.

The treatment employs a modified, non-disease-causing virus vector to deliver RNA interference molecules into brain cells, aiming to lower toxic huntingtin protein production. This approach involves precise neurosurgical infusion, distinguishing it from systemic treatments.

UniQure submitted a marketing application to the U.S. Food and Drug Administration (FDA) in early September, with the agency agreeing to review the therapy based on three-year data. An application has also been filed with the United Kingdom’s Medicines and Healthcare products Regulatory Agency. The company operates facilities in Amsterdam and Lexington, Massachusetts.

Huntington’s disease affects approximately 75,000 people in the U.S., Europe, and the UK, and currently there are no approved therapies that slow its progression. Experts have cautioned that the study’s small sample size and reliance on a historical control group may limit the conclusiveness of the results, but many agree the data represent a meaningful advance in treating this fatal inherited condition.

Following the updated results, UniQure’s share price declined by 37 percent, reflecting investor concerns about the apparent reduction in treatment effect over time. Nonetheless, the therapy’s ability to slow functional decline and impact biological markers is regarded as unprecedented by researchers in the field. Further evaluation by regulatory authorities is ongoing.

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