Archive · 26 Sep 2025
Huntington's Disease Successfully Treated With Gene Therapy
Scientists have made a major breakthrough in treating Huntington's disease.
They used a new type of treatment called gene therapy.
This treatment uses a special delivery system, like a virus, to deliver a gene payload directly into the brain.
The treatment works by silencing the faulty gene that causes the disease.
Patients who received the treatment showed a significant slowing of the disease's progression.
This new treatment is the first to show indications of changing the course of the disease, offering hope for patients and families.
While the results are promising, more research is needed to confirm safety and long-term effectiveness.
Researchers used gene therapy to treat Huntington's disease.
The treatment is called AMT-130.
The treatment reduced disease progression by almost 75% in the high-dose group.
The treatment involves a single injection into the brain.
Researchers plan to apply for regulatory approval as early as 2026.
- Who
- Researchers at University College London and the biotech company uniQure.
- What
- Successfully treated Huntington's disease using experimental gene therapy.
- When
- Trial conducted over three years.
- Where
- University College London.
- Why
- To halt the progression of Huntington's disease and improve patient outcomes.
- Treatment Name
- AMT-130
- Disease
- Huntington's disease
- Trial Location
- University College London
- Results
- Reduced disease progression by ~75% in high-dose group over 3 years
- Treatment Method
- Single surgical injection into the brain
- Company
- uniQure
- Application for approval
- as early as 2026