uniQure N.V. (NASDAQ: QURE) is a leading gene therapy company focused on developing transformative gene therapies for patients with severe medical needs. Based in Lexington, Massachusetts, with European operations in Amsterdam, uniQure has developed a proprietary modular gene therapy platform that enables efficient delivery and sustained expression of therapeutic proteins. The company is a pioneer in AAV (adeno-associated virus) gene therapy, with the first gene therapy for hemophilia B (Hemgenix/etranacogene dezaparvovec) approved in the United States and Europe.
| Attribute |
Details |
| Headquarters |
Lexington, Massachusetts, USA |
| European Office |
Amsterdam, Netherlands |
| Founded |
2001 |
| Stock Symbol |
QURE (NASDAQ) |
| Market Cap |
~$500M |
| Employees |
~500 |
| CEO |
Matt Kapusta |
- 2001: Founded in Amsterdam, Netherlands
- 2014: Went public on NASDAQ
- 2019: Established US headquarters in Lexington, MA
- 2020: Advanced Huntington's disease program into clinical trials
- 2022: Received FDA approval for Hemgenix (Hemophilia B)
- 2023: Expanded CNS pipeline with new AD/PD programs
- 2024: Continued advancement of Huntington's program
- 2025: Expanded manufacturing capacity
- Indication: Hemophilia B
- Mechanism: AAV5-delivered Factor IX gene therapy
- Approval: FDA (November 2022), EMA (2023)
- Price: $3.5M per dose (most expensive drug in the world)
- Clinical Data: Demonstrated sustained Factor IX expression with reduced bleeding rates
uniQure has a diversified pipeline spanning hematology and CNS disorders:
| Drug |
Indication |
Mechanism |
Stage |
Status |
| AMT-130 |
Huntington's disease |
miRNA targeting huntingtin |
Phase 1/2 |
Enrolling |
| AMT-162 |
Amyotrophic lateral sclerosis |
SOD1 silencing |
Preclinical |
Active |
| AMT-240 |
Alzheimer's disease |
Apolipoprotein E modulation |
Preclinical |
Active |
| AMT-260 |
Parkinson's disease |
GBA gene therapy |
Discovery |
Research |
| AMT-280 |
Spinal cord injury |
Nogo-A inhibition |
Discovery |
Research |
| Drug |
Indication |
Mechanism |
Stage |
Status |
| Etranacogene dezaparvovec (Hemgenix) |
Hemophilia B |
Factor IX gene therapy |
Approved |
Commercial |
| AMT-180 |
Hemophilia A |
Factor VIII gene therapy |
Preclinical |
Active |
| AMT-191 |
Hemophilia A (inhibitors) |
Novel approach |
Discovery |
Research |
uniQure has developed a comprehensive AAV gene therapy platform:
- Proprietary AAV Capsids: Next-generation capsids with enhanced CNS delivery
- Tissue-Specific Promoters: Engineered promoters for targeted expression
- Self-Complementary Vectors: Optimized for rapid onset of expression
- In-House GMP Facility: State-of-the-art manufacturing in Lexington, MA
- Scalable Platform: Suspension cell culture for large-scale production
- Quality Control: Integrated analytical and quality systems
- CNS Delivery: AAV9 and novel capsids for brain delivery
- Peripheral Delivery: Established delivery to liver and other tissues
- Novel Routes: Exploration of intrathecal and intravenous delivery
AMT-130 is uniQure's most advanced CNS program:
- Approach: AAV5-delivered microRNA (miHTT) to silence mutant huntingtin
- Target: Reduces production of mutant huntingtin protein
- Delivery: Direct intracerebral injection
- Phase 1/2 Trial: Ongoing in US and Europe
- Primary Endpoints: Safety and tolerability
- Secondary Endpoints: Biomarker reduction, clinical efficacy
- Dosing: Single administration with sustained effect
- Demonstrated dose-dependent reduction of mutant huntingtin protein in CSF
- Generally well-tolerated with manageable safety profile
- Early signs of potential clinical benefit
uniQure is developing a novel approach for Alzheimer's disease:
- Apolipoprotein E (APOE): Key genetic risk factor for late-onset AD
- Approach: Modulate APOE expression to reduce AD risk
- Gene therapy to deliver protective APOE variants
- AAV-mediated delivery to CNS
- Potential disease-modifying approach
¶ Competitive Landscape
uniQure competes in the gene therapy space with:
- BioMarin Pharmaceutical: Hemophilia gene therapy
- Pfizer: Hemophilia gene therapy programs
- Spark Therapeutics (Roche): Hemophilia and CNS programs
- Sangamo Therapeutics: Gene therapy platform
- Voyager Therapeutics: CNS gene therapy programs
- Lexington, MA: Primary manufacturing facility (100,000+ sq ft)
- Amsterdam, EU: Process development and analytics
- Capacity: Multiple 2000L bioreactor trains
- FDA and EMA approved manufacturing
- Full GMP compliance
- Integrated quality management
- Exchange: NASDAQ
- Symbol: QURE
- IPO: 2014
- Hemgenix Sales: Commercial revenues from Hemophilia B therapy
- Partnerships: Strategic collaborations with major pharmaceutical companies
- CSL Behring: Hemophilia B partnership (Hemgenix commercialization)
- Bristol-Myers Squibb: CNS research collaboration (historical)
- Genentech: Huntington's disease collaboration (historical)
uniQure maintains an active publication record: