UniQure NV - Company Profile

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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.

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Headquarters Netherlands

Address Paasheuvelweg 25a, Amsterdam, Noord-Holland, 1105BP


Telephone 31 202 406000

No of Employees 209

Industry Pharmaceuticals and Healthcare

Ticker Symbol & Exchange QURE (NASD)

Revenue (2024) $16.1M -40.6% (2024 vs 2023)

EPS XYZ

Net Income (2024) XYZ 16.9% (2024 vs 2023)

Market Cap* $1.6B

Net Profit Margin (2024) XYZ -39.9% (2024 vs 2023)

* As of and is in US$
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UniQure NV premium industry data and analytics

20+

Pipeline Drugs

Identify which of UniQure NV’s products will be commercialized, helping you to better contend with shifting product portfolios, and allowing you to stay one step ahead of the market.

20+

Catalyst Calendar

Proactively evaluate UniQure NV’s catalyst impacts to stay ahead of the competition & improve corporate planning. Bolster business development with timely opportunities.

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Clinical Trials

Determine UniQure NV go-to-market proposition and gain insight into the company’s clinical operations, recruitment, and trial strategy.

12+

Insights

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6

Regulatory Milestones

Track drug and patent expiries along with geographies impacted.

1

Marketed Drugs

Understand UniQure NV’s commercialized product portfolio to stay one step ahead of the market.

1

ICT Spend & Tech Priorities

IT Client Prospector provides intelligence on UniQure NV’s likely spend across technology areas enabling you to understand the digital strategy.

Products and Services

Products Brands
Etranacogene Dezaparvovec: Hemophilia B miQURE
AMT-130: Huntington’s Disease GoQURE
AMT-260: Temporal Lobe Epilepsy LinQURE
XYZ
XYZ
XYZ
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History

History section provides information on new products, mergers, acquisitions, expansions, approvals, and many more key events.

Year Event Description
2025 Regulatory Approval In April, the company announced that the US Food and Drug Administration (FDA) had granted breakthrough therapy designation to AMT-130 for the treatment of Huntington’s disease.
2024 Contracts/Agreements In December, the company signed an agreement with the U.S. Food and Drug Administration for an Accelerated Approval pathway for AMT-130.
2024 Regulatory Approval In September, the company announced orphan drug designation had been granted to AMT-191 for the treatment of Fabry disease by the US FDA.
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Competitor Comparison

Key Parameters UniQure NV Roche Holding AG Pfizer Inc Sanofi Takeda Pharmaceutical Co Ltd
Headquarters Netherlands Switzerland United States of America France Japan
City Amsterdam Basel New York City Paris Chuo-Ku
State/Province Noord-Holland Basel-Stadt New York Ile-de-France Tokyo
No. of Employees 209 112,774 75,000 76,493 47,455
Entity Type Public Public Public Public Public
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Executives
Name Position Board Since Age
Matthew Kapusta Chief Executive Officer; Director Executive Board 2016 52
Christian Klemt Chief Financial Officer Senior Management 2021 52
Walid Abi-Saab, MD Chief Medical Officer Senior Management 2023 59
Richard Porter Chief Business Officer; Chief Scientific Officer Senior Management 2023 -
Amin Abujoub, PhD Chief Technical Operations Office Senior Management 2024 -
Non Dignissim Eros Proin vel Convallis 2026 XY
Non Dignissim Eros Proin vel Convallis 2026 XY
Non Dignissim Eros Proin vel Convallis 2026 XY
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