Experimental Gene Therapy Successfully Treats Huntington’s Disease for the First Time

Preliminary results from a small study offer hope for treatment of the degenerative disease

Sara Hashemi | Daily Correspondent

September 25, 2025

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An experimental gene therapy has slowed the progression of Huntington’s disease by 75 percent, according to results reported by the biotechnology company uniQure this week.

Huntington’s is an inherited degenerative disease that is caused by a mutation in a gene called HTT. This mutation impacts the huntingtin protein, causing a toxic form of it that causes cognitive decline, involuntary movements, slurred speech and other symptoms. The disease generally presents itself in patients in their 30s and 40s, and impacts about 30,000 people in the United States.

“We now have a treatment for one of the world’s more terrible diseases. This is absolutely huge. I’m really overjoyed,” says Sarah Tabrizi, who led the trial and is the director of University College London’s Huntington’s disease centre, to Hannah Devlin at the Guardian.

The treatment, called AMT-130, targets the mutated protein. In the trial, 29 participants received a shot of the drug to their brains during a 12- to 20-hour surgical procedure. The shot sends a harmless virus encoded with special instructions to turn off the production of the mutated protein.

AMT-130 Administration

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Of the 29 participants, 17 received a high dose of the treatment, while 12 participants received a low dose. Twelve patients from each group were then followed over the course of 36 months, and their progress was compared to that of about 1,600 patients with Huntington’s at a similar disease stage who were not receiving treatment.

The researchers used the Unified Huntington’s Disease Rating Scale to track everyone’s symptoms. Those who received the high dosage of the drug experienced 75 percent less disease progression over the course of study. They also had an 8.2 percent reduction in neurofilament light, a biomarker for dying neurons associated with Huntington’s disease.

“The clinical results are very encouraging,” says Ole Isacson, a neuroscientist at Harvard University who was not involved in the work, to Jocelyn Kaiser at Science.

UniQure will submit its data to the Food and Drug Administration later this year and hopes to gain FDA approval to launch the product by 2027. But it’s important to note that this was a small trial, and the results were not published in a peer-reviewed journal. More testing is also needed to better understand the treatment’s side effects. Further monitoring will also show if participants maintain their progress over time.

Victor Sung, a principal investigator for the trial and a movement disorders neurologist at the University of Alabama at Birmingham, tells Carolyn Y. Johnson at the Washington Post that the treatment will also likely be expensive and will require surgery at specialized centers.

Still, the participants’ lives have already been improved thanks to the treatment. “This result changes everything,” says Ed Wild, a neuroscientist at University College London and principal investigator in the work, in a press release. “My patients in the trial are stable over time in a way I’m not used to seeing in Huntington’s disease—and one of them is my only medically retired Huntington’s disease patient who has been able to go back to work.”

Sara Hashemi | Read More

Sara Hashemi is a science writer and fact-checker currently based in New York City. Her work has appeared in Sierra, The Body, Maisonneuve magazine and more.