AMT-130 gene therapy reduces Huntington's progression about 75% in trial
Scientists have developed a new gene therapy called AMT-130 that might actually slow down Huntington's disease, which has had no disease-modifying treatment until now.
After 30 years of searching, this therapy targets the root genetic mutation and cuts down the harmful protein behind the disease.
In early trials with 29 people, those in the dose group who got AMT-130 saw about 75% less progression of Huntington's disease than the control group, plus fewer signs of brain damage.
The treatment is delivered right to the part of the brain most affected.
FDA grants AMT-130 expedited status
If approved, AMT-130 would be the first treatment to actually change how Huntington's unfolds, not just manage symptoms.
The FDA has already given it special status to speed things up, and UniQure planned to seek approval in early 2026.
For families facing Huntington's with no real options so far, this could mean genuine hope for a better future.