UniQure

uniQure

Company type	Public
Traded as	Nasdaq:  QURE
Industry	Pharmaceutical Medical Technology
Website	uniqure.com

uniQure is a Dutch biopharmaceutical company which makes gene therapies. It has developed several adeno-associated virus gene therapies.

History

In 2012 uniQuire won approval for alipogene tiparvovec (trade name Glybera) for treatment of lipoprotein lipase deficiency. The drug came off the market in 2017 due to limited demand largely blamed on its over US$1 million cost. ^[1]

In 2022, the US Food and Drug Administration (FDA) granted approval to uniQure's etranacogene dezaparvovec (trade name Hemgenix) for treatment of haemophilia B. The drug was the fifth gene therapy for a rare genetic disease to ever win approval. ^[2] uniQure partnered with CSL Behring to commercialize Hemgenix. ^[3]

In late 2025, the FDA reversed an eariler decision to grant breakthrough status to uniQure's investigational drug for Huntington's disease, AMT-103. ^{[4] [5] [6] [7]}

References

1. Crowe K. "The million-dollar drug". CBC News. Retrieved 14 November 2025.
2. Mullard, Asher. "FDA approves first haemophilia B gene therapy". Nature (journal) . doi:10.1038/d41573-022-00199-8 . Retrieved 14 November 2025.
3. Taylor, Nick (25 June 2025). "CSL to pay $450M to buy uniQure's hemophilia B gene therapy". FierceBiotech . Retrieved 14 November 2025.
4. Santhosh, Christy (3 November 2025). "UniQure shares plummet as FDA says inadequate clinical data for Huntington's therapy". Reuters . Retrieved 14 November 2025.
5. "uniQure Staggers as FDA Questions Data for Huntington's Gene Therapy Candidate". Genetic Engineering and Biotechnology News . 3 November 2025. Retrieved 14 November 2025.
6. Pernell, Avalon (3 November 2025). "UniQure Tunmbles After 'Surprising' Pivot by FDA on Drug Data". Bloomberg News . Retrieved 14 November 2025.
7. "Artificial miRNA slows Huntington's". Nature (journal) . doi:10.1038/s41587-025-02910-7 . Retrieved 14 November 2025.