Huntington’s disease is a rare, inherited neurodegenerative disorder that affects the brain and drastically decreases quality of life. Although Huntington’s disease has been present for centuries, it was successfully treated for the first time, in September of 2025, by the company UniQure using a treatment called AMT-130.
Huntington’s disease is caused by a mutation in the HTT gene, which is responsible for producing a protein called huntingtin. In people with Huntington’s disease, the HTT gene contains an abnormal repetition of CAG sequences, leading to a toxic form of the huntingtin protein accumulating in the brain. This buildup causes a variety of symptoms, such as loss of coordination, slurred speech, mood swings and variation in lifespan.
“On average, individuals suffering from Huntington’s disease may live up to 10 to 20 years depending on CAG repeats, but everyone’s course is different and variable,” said Dr. Kelly Andrzejewski, a neurologist who serves as director of UB’s Huntington Disease society.
Aware of the detrimental effects of this disease, researchers at the University College London Huntington’s Disease Centre were committed to finding an effective treatment and improving the quality of life for those suffering from it. Through hard work and countless trials, professors Ed Wild and Sarah Tabrizi were astonished to learn that the treatment they led in London was responsible for significantly slowing disease progression. The treatment is a form of gene therapy administered through 12 to 18 hours of meticulous brain surgery.
The procedure begins with a safe virus engineered to contain a specific sequence of DNA. This DNA sequence is infused deep into the brain, specifically into the putamen and caudate nucleus, where it produces microRNA that permanently blocks the production of mutant huntingtin.
Remarkably, AMT-130 reduced the progression of Huntington’s disease symptoms by 75 percent over three years in the 12 participants who received the treatment. It also decreased neurodegeneration within the spinal fluid.
“This breakthrough gives hope to patients and may lead to further research being done to improve treatment further,” said Pretesh Prakash, a junior neuroscience major.
Those who received the treatment were ecstatic with the results and hopeful for a future where their disease no longer defines them. UniQure, the governing body of this treatment, announced plans to submit a biologics license application to the U.S. Food and Drug Administration in 2026, hoping to make this treatment readily available to individuals across the globe.
AMT-130 has proved to be revolutionary, but has its fair share of concerns. Gene therapy comes with a hefty price tag, which may pose payment difficulties and access barriers for patients. In addition to this, AMT-130 tends to be invasive, complex, intricate and lengthy, which will possibly be a deterrent for future patients. Furthermore, once the gene therapy is administered, it’s impossible to remove or reverse — which may be too much of a commitment for some.
Nonetheless, this breakthrough is still in its early stages and consists of many moving parts, but it has motivated aspiring medical professionals.
“As a future doctor, I am inspired because we are now seeing decades-long research come together to improve patient life satisfaction,” said Andrea Calderon, a senior biological sciences major.
Though numerous advancements in healthcare have resulted in a healthier population, Dr. Andrzejewski believes that remaining educated is a necessity.
“It’s important for everyone to be aware and knowledgeable about their health, as well as the diseases that affect society,” said Dr. Andrzejewski.
This groundbreaking treatment marks a pivotal step toward managing Huntington’s disease and offers new hope for patients, families and future generations impacted by the condition.
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