Some patients who took part in early-stage clinical trials at University College London (UCL) saw the speed at which their condition developed reduced by 75% after three years, according to uniQure, a gene therapy company based in the Netherlands and the United States.
The study tested a new gene therapy, AMT-130, which is delivered through an injection directly into the brain.
Researchers said that AMT-130 works by permanently introducing new functional DNA into a patient’s cells.
“This result changes everything,” said the lead researcher, Ed Wild of UCL’s Huntington’s Disease Center.