uniQure N.V. (QURE) Reports Positive Phase I/II Results, Prepares FDA BLA Submission

We recently compiled a list of the 12 Best Performing Healthcare Stocks to Buy Right Now. uniQure N.V. is one of them.

uniQure N.V. (NASDAQ:QURE) is a leading gene therapy company focused on developing transformative treatments for severe medical conditions. Its pipeline includes HEMGENIX for hemophilia B and AMT-130, a gene therapy candidate for Huntington’s disease, which has recently attracted significant attention.

In late September 2025, uniQure N.V. (NASDAQ:QURE) announced positive topline results from pivotal Phase I/II trials of AMT-130, showing a 75% slowing of disease progression over three years. These results mark a major milestone and set the stage for a Biologics License Application (BLA) submission to the FDA in early 2026, aiming for near-term market approval.

To support development and commercialization, the business raised $300 million through a public offering and secured a $175 million senior secured term loan. Alongside AMT-130, QURE continues to advance other gene therapy candidates, including AMT-260 for mesial temporal lobe epilepsy, AMT-162 for ALS, and AMT-191 for Fabry disease, highlighting a diversified pipeline targeting rare diseases.

Analysts have responded positively to the firm’s clinical breakthroughs and financial backing, with strong buy ratings and optimistic price targets. The combination of groundbreaking AMT-130 data, robust funding, and a broad pipeline positions uniQure N.V. (NASDAQ:QURE) among the best performing stocks in the healthcare sector, offering potential transformative impact for patients and promising near-term growth for investors.

While we acknowledge the potential of QURE as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock.

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