For generations, families affected by Huntington’s disease have faced a heartbreaking reality: no medication or doctor could slow its devastating march through the brain. The disease robs people of movement, memory, and independence, often striking in the prime of their life.
Now, for the first time, scientists say that decline may finally be slowed.
On September 24, uniQure, a biotech company based in Amsterdam, announced the results of a major clinical trial it had conducted. Its experimental gene therapy, AMT-130, was shown to have significantly slowed the progression of Huntington’s disease. In a news release that same day, uniQure wrote that patients who received a high dose of the therapy lost motor, cognitive, and psychiatric abilities about 75% slower than those who didn’t receive the treatment.
“This pivotal study of AMT-130 showed statistically significant effects on both cUHDRS and TFC at 36 months,” Dr. Sarah Tabrizi, director of the Huntington’s Disease Center at University College London, said in a statement, according to NeurologyLive. The cUHDR stands for composite Unified Huntington’s Disease Rating Scale and is used as a comprehensive assessment tool in clinical trials to measure the progression of Huntington’s. TFC stands for Total Functioning Capacity and is a part of the cUHDRS, serving as one of several assessment tools within the comprehensive scale to evaluate the clinic features of Huntington’s disease. “I believe these groundbreaking data are the most convincing in the field to date and underscore potential disease-modifying effects in Huntington’s disease,” Tabrizi said.
Huntington’s disease is caused by a genetic mutation in the HTT gene that makes the body produce a faulty version of the Huntingtin protein, which accumulates in a part of the brain called the striatum and causes nerve cells in the brain to decay over time. This causes symptoms such as uncontrolled movements, difficulty speaking and swallowing, impaired judgment, and memory issues. Symptoms usually begin in a person’s 30s or 40s and worsen over time, leading to problems walking, speaking, and thinking clearly. Children of those who have Huntington’s disease have a 50% chance of inheriting it.
AMT-130 takes a new approach. It works by using adeno-associated viruses to produce microRNA that targets and breaks down the mRNA that carries blueprints for the faulty huntingtin proteins. According to Scientific American, doctors deliver the therapy by inserting catheters into parts of the brain, such as the striatum, allowing them to administer the drug directly to the neurons (brain cells) that produce the faulty proteins.
This sets AMT-130 apart from older experimental drugs known as ASOs, which also target mRNA but require repeated spinal injections several times a year. Those earlier approaches produced mixed results, and in some cases even did more harm than good. In contrast, uniQure’s therapy appears to provide long-term benefits after just a single procedure.
In addition to slowing disease progression, patients receiving AMT-130 maintained stronger thinking and motor skills. Key biomarkers of brain health improved as well: levels of neurofilament light protein, which rises when neurons are dying, dropped below baseline after 36 months, suggesting reduced ongoing damage. The treatment seems generally safe: since 2022, there have been no new serious drug-related side effects, according to Huntington’s Disease News.
“These findings reinforce our conviction that AMT-130 has the potential to fundamentally transform the treatment landscape for Huntington’s disease,” Dr. Walid Abi-Saab, uniQure’s chief medical officer, said, according to CNN.
UniQure plans to apply for a US licence in early 2026. If the FDA grants approval, AMT-130 could reach American patients as soon as late 2026, as stated by BBC. The therapy has already earned Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations, both of which are reserved for promising treatments that may address serious or life-threatening diseases.
Huntington’s disease affects about 75,000 people in the United States and Europe, with many more carrying the gene that could one day cause it. For those families, the results from AMT-130 offer something that has long been out of reach: hope.
