UniQure shares fall after mixed long-term Huntington’s gene-therapy data

  • UniQure released four-year AMT-130 data from 12 high-dose Huntington’s disease patients.
  • UniQure shares opened 39% lower on September 29 after the long-term data release.
  • UniQure submitted a September 2 BLA based on its three-year AMT-130 analysis.

UniQure lost about $1 billion in market value after releasing updated long-term data for AMT-130, its experimental gene therapy for Huntington’s disease. Shares opened 39% lower on September 29, 2026, after the 48-month results showed a smaller treatment benefit than the three-year data. The investor notice described the four-year result as a 44% slowing of progression on cUHDRS, compared with 75% at 36 months, while other reporting on the company’s update cited 44% versus 80% and a 61% four-year slowing on Total Functional Capacity. The four-year cUHDRS result did not reach statistical significance. UniQure submitted a Biologics License Application on September 2 based on the three-year analysis, and SueWallSt, powered by Levi & Korsinsky LLP, announced an investigation into whether earlier statements about AMT-130’s clinical results and durability were materially misleading. UniQure has not been established to have violated securities laws.

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