Huntington’s disease progression may be slowed by experimental gene therapy

There may be some hope for those living with Huntington’s disease after a new experimental...
There may be some hope for those living with Huntington’s disease after a new experimental gene therapy was reportedly found to slow the progression of the disease.(Source: CNN/POOL)
Published: Sep. 25, 2025 at 12:33 AM EDT

(CNN) - There may be some hope for those living with Huntington’s disease after a new experimental gene therapy was reportedly found to slow the progression of the disease.

Drugmaker Uniqure announced the results of the early-stage trial Wednesday. The trial included 29 people with Huntington’s, an inherited progressive brain disorder, who were treated with either low or high doses of the gene therapy.

Uniqure says the patients who received the high dose saw disease progression slow by 75% after three years. The treatment was generally well-tolerated, according to the company.

The drugmaker says it plans to submit its data to the Food and Drug Administration for approval in the first quarter of next year.

There is currently no treatment that can cure, stop or reverse Huntington’s, but some of the symptoms can be treated.

It’s important to note Uniqure’s study results have not been published or peer-reviewed.