UniQure to unveil four-year data on Huntington's gene therapy AMT-130
UniQure will release four-year results for its AMT-130 gene therapy targeting Huntington’s disease before the end of September.
The biotech firm plans to publish the four-year follow-up of its AMT-130 trial, which treats Huntington’s disease. Earlier, three-year data showed a 75% slowdown in disease progression for the high-dose group of 12 patients compared with a matched external control cohort. The upcoming readout is expected before September ends.
Why it matters
The results could confirm a disease-modifying treatment for Huntington’s, a condition with limited therapeutic options.
In this story
AMT-130Huntington’s diseasegene therapyfour-year resultsclinical trialdisease progressionexternal control groupnatural history study
