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Highlights
UniQure NV (UniQure) is a biotechnology company that researches, develops, and commercializes gene therapies for rare and severe diseases using an adeno-associated virus (AAV)-based technology platform. The company's pipeline of innovative gene therapies focuses on the treatment of Huntington’s disease, amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD 1), refractory mesial temporal lobe epilepsy (mTLE), and Fabry disease. UniQure’s lead product, AMT-060, is a gene therapy for the treatment of moderately-severe hemophilia B. The company, which distributes etranacogene dezaparvovec, is used to treat Hemophilia B. Its preclinical products include AMT-130, which focuses on the treatment of Huntington disease; AMT-260, to treat Temporal Lobe Epilepsy; AMT-191, to treat Fabry disease; and AMT-162, to treat ALS (SOD1). It has operations in the US, the Netherlands, and Switzerland. UniQure is headquartered in Amsterdam, the Netherlands.UniQure NV Key Recent Developments
- Apr 28, 2026: uniQure to Report First Quarter 2026 Financial Results
- Mar 31, 2026: Hagens Berman Updates uniQure Investigation After FDA Rebukes and Allegations of “Distorted” Data
- Mar 30, 2026: Deadline to Join Class Action Against uniQure N.V. (QURE)
- Mar 11, 2026: FDA Reportedly Accuses uniQure of Pushing 'Distorted' and 'Manipulated' Data for 'Failed' AMT-130 Drug Amid Securities Class Action
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Table of Contents
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- Sangamo Biosciences
- NeuExcell Therapeutics Inc
- CANbridge Pharmaceuticals Inc
- Wave Life Sciences Ltd
- Regeneron Pharmaceuticals Inc
- Skyhawk Therapeutics Inc
- Prilenia Therapeutics BV
- Lexeo Therapeutics Inc
- Biogen Inc
- AskBio GmbH
- Adverum Biotechnologies Inc
- Sanofi
- Neurona Therapeutics Inc
- Ionis Pharmaceuticals Inc
- Idorsia Pharmaceutical Ltd
- Expression Therapeutics Inc
- Spur Therapeutics Ltd
- Passage Bio Inc
- Intellia Therapeutics Inc
- BioMarin Pharmaceutical Inc
- PTC Therapeutics Inc
- Novartis Gene Therapies
- Astellas Gene Therapies
- Annexon Inc
- Alnylam Pharmaceuticals Inc
- 4D Molecular Therapeutics Inc
- Voyager Therapeutics Inc
- Pfizer Inc
- EpiBlok Therapeutics GmbH
- Amicus Therapeutics Inc
- Takeda Pharmaceutical Co Ltd
- Roche Holding AG
- Neurimmune Holding AG
- Genetix Biotherapeutics Inc
- Abeona Therapeutics Inc

