High-dose AMT130 gene therapy slows Huntington's progression

Researchers in London and the Netherlands reported Wednesday that an experimental, one-time gene therapy markedly slowed progression of Huntington's disease in an early-stage trial, a result that could pave the way for the first licensed disease-modifying treatment for the fatal neurodegenerative condition. UniQure, the gene therapy company that developed the treatment, and investigators at University College London said patients given the highest dose of AMT130 experienced about 75 percent less clinical decline over 36 months compared with an external control group drawn from a large natural history study.

In the three-year analysis, patients treated with the high dose of AMT130 lost an average of 0.38 points on a composite clinical measure known as cUHDRS, compared with a 1.52 point decline among matched participants in the external control cohort, uniQure said. The benefit was statistically significant and met the trial's primary endpoint, the company added. Twenty-nine patients enrolled in the trial, and the 12 who received the highest dose showed the greatest slowing of disease progression, investigators said.

AMT130 is delivered by direct injection into the brain through...