uniQure N.V.
uniqure.comWhat Do They Do?
Develops gene therapies for patients with rare and other serious diseases, including Huntington’s disease, refractory temporal lobe epilepsy and Fabry disease.
10-K for FY2025, filed Mar 2, 2026We are a leader in the field of gene therapy, seeking to deliver to patients suffering from rare and other devastating diseases single…View ↗
Who Are Their Customers?
CSL Behring is the customer for HEMGENIX; it holds the therapy’s exclusive global rights under a license agreement.
10-K for FY2025, filed Mar 2, 2026In June 2020 the Company entered into the CSL Behring Agreement pursuant to which CSL Behring received the exclusive global rights to…View ↗
Do Revenues Turn Into Profits?
No. It lost $199M in FY2025.
How Are They Changing?
- Net income
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Sources
10-Q for Q2 FY2026, filed Jul 29, 2026
−$81.1MView ↗
- ▲ +115% (Loss)
- vs −$37.7M in Q2 FY2025
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Sep 2, 2026
Filed U.S. and U.K. applications for AMT-130
The applications seek accelerated approval in the United States and marketing authorization in the United Kingdom.
Sources
8-K, filed Sep 2, 2026
uniQure Announces Submission of Biologics License Application for Ifezuntirgene Inilparvovec (AMT-130) in Huntington’s DiseaseView ↗
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Jun 17, 2026
FDA said existing AMT-130 data could support an accelerated-approval application
The FDA sought alignment on the confirmatory study design before the application, including consideration of a standard-of-care control.
Sources
8-K, filed Jun 17, 2026
Lexington, MA and Amsterdam, the Netherlands, June 17, 2026 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing…View ↗
HEMGENIX production moved out
The older report said uniQure manufactured HEMGENIX for CSL Behring; the latest says Genezen provides it directly to CSL Behring and uniQure arranges that supply.
10-K for FY2022, filed Feb 27, 2023 10-K for FY2025, filed Mar 2, 2026We are a leader in the field of gene therapy and seek to deliver to patients suffering from rare and other devastating diseases single…View ↗
In the period between April 2022 and July 2024, the Company manufactured HEMGENIX for CSL Behring in accordance with a June 2020…View ↗
Programs advanced to clinical development
Temporal lobe epilepsy and Fabry programs shifted from preclinical candidates to clinical candidates.
10-K for FY2022, filed Feb 27, 2023 10-K for FY2025, filed Mar 2, 2026We are a leader in the field of gene therapy and seek to deliver to patients suffering from rare and other devastating diseases single…View ↗
We are a leader in the field of gene therapy, seeking to deliver to patients suffering from rare and other devastating diseases single…View ↗
Who Do They Compete With?
Competes with Novartis, Roche, Wave Life Sciences, Alnylam Pharmaceuticals, Regeneron Pharmaceuticals, Skyhawk Therapeutics, Sarepta Therapeutics, Neurona Therapeutics, EpilepsyGTx, Xenon Pharmaceuticals, Biogen, Ionis Pharmaceuticals, Neurimmune, Coya Therapeutics, Amylyx Pharmaceuticals, NeuroSense Therapeutics, AL-S Pharma, Voyager Therapeutics, Amicus Therapeutics, and Sanofi
10-K for FY2025, filed Mar 2, 2026Our key competitors, focused on developing therapies in various indications, include among others, Novartis, Roche, Wave Life Sciences…View ↗
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