UniQure's gene therapy AMT-130 slowed Huntington’s progression by 75% in a trial, boosting stock over 230%.

UniQure's stock jumped over 230% to around $45.60 on Wednesday after its gene therapy AMT-130 met primary and secondary endpoints in a Phase I/II trial for Huntington’s disease, showing a 75% slowing of disease progression at 36 months and a 60% reduction in functional decline. The therapy was well-tolerated, with positive trends in motor and cognitive measures and reduced neurodegeneration biomarkers. The company plans to file a Biologics License Application in early 2026 and has secured a $175 million non-dilutive loan from Hercules Capital to support commercial readiness.