UniQure NV (NASDAQ: QURE) just staged one of biotech's wildest rides of the year, soaring 248% in a single session after unveiling successful trial results for its experimental Huntington's disease gene therapy.
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uniQure (NASDAQ: QURE) has been one of the most eye-catching movers in recent sessions. The stock surged ~250% this Wednesday after reports that the company's gene therapy showed positive results in treating Huntington's disease.
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UniQure said the high-dose of its AMT-130 therapy slowed Huntington’s disease progression by up to 75% over three years and plans to seek FDA approval in early 2026.
UniQure N.V. (NASDAQ:QURE) shares are trading higher Wednesday after the company announced positive topline results from Phase I/II study of AMT-130 in patients with Huntington's Disease.
UniQure's AMT-130 gene therapy slowed Huntington's disease progression in a Phase 1/2 trial, supported by new $175 million financing for launch readiness.
The company stated that high-dose AMT-130 demonstrated 75% disease slowing at 36 months, as measured by the composite Unified Huntington's Disease Rating Scale.
uniQure's early AMT-191 trial data in Fabry disease showed strong enzyme activity, manageable safety, and patient independence from enzyme replacement therapy.
FDA launches Rare Disease Evidence Principles to guide therapy reviews, offering new pathways for approval while raising questions on development impact.
