uniQure N.V.
Overview
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companies_uniqure_1["History and Milestones"]
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companies_uniqure_2["FDA-Approved Products"]
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companies_uniqure_3["Hemgenix Etranacogene Dezaparvovec"]
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companies_uniqure_4["Pipeline Overview"]
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companies_uniqure_5["CNS/Neurology Programs"]
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uniQure N.V.
Overview
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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["@uniqure"]. 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.
Company Profile | Attribute | Details |
History & Milestones
2001 : Founded in Amsterdam, Netherlands2014 : Went public on NASDAQ2019 : Established US headquarters in Lexington, MA2020 : Advanced Huntington's disease program into clinical trials2022 : Received FDA approval for Hemgenix (Hemophilia B)2023 : Expanded CNS pipeline with new AD/PD programs2024 : Continued advancement of Huntington's program2025 : Expanded manufacturing capacityFDA-Approved Products
Hemgenix (Etranacogene Dezaparvovec)
Indication : Hemophilia BMechanism : AAV5-delivered Factor IX gene therapyApproval : 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 ratesPipeline Overview uniQure has a diversified pipeline spanning hematology and CNS disorders:
CNS/Neurology Programs | Drug | Indication | Mechanism | Stage | Status |
Hematology Programs | Drug | Indication | Mechanism | Stage | Status |
uniQure has developed a comprehensive AAV gene therapy platform:
Vector Technology
Proprietary AAV Capsids : Next-generation capsids with enhanced CNS deliveryTissue-Specific Promoters : Engineered promoters for targeted expressionSelf-Complementary Vectors : Optimized for rapid onset of expressionManufacturing
In-House GMP Facility : State-of-the-art manufacturing in Lexington, MAScalable Platform : Suspension cell culture for large-scale productionQuality Control : Integrated analytical and quality systemsDelivery
CNS Delivery : AAV9 and novel capsids for brain deliveryPeripheral Delivery : Established delivery to liver and other tissuesNovel Routes : Exploration of intrathecal and intravenous deliveryHuntington's Disease Program (AMT-130) AMT-130 is uniQure's most advanced CNS program:
Mechanism
Approach : AAV5-delivered microRNA (miHTT) to silence mutant huntingtinTarget : Reduces production of mutant huntingtin proteinDelivery : Direct intracerebral injectionClinical Development
Phase 1/2 Trial : Ongoing in US and EuropePrimary Endpoints : Safety and tolerabilitySecondary Endpoints : Biomarker reduction, clinical efficacyDosing : Single administration with sustained effectClinical Data
Demonstrated dose-dependent reduction of mutant huntingtin protein in CSF
Generally well-tolerated with manageable safety profile
Early signs of potential clinical benefit
Alzheimer's Disease Program (AMT-240) uniQure is developing a novel approach for Alzheimer's disease:
Target
Apolipoprotein E (APOE) : Key genetic risk factor for late-onset ADApproach : Modulate APOE expression to reduce AD riskMechanism
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 therapyPfizer : Hemophilia gene therapy programsSpark Therapeutics (Roche): Hemophilia and CNS programsSangamo Therapeutics : Gene therapy platformVoyager Therapeutics : CNS gene therapy programsManufacturing & Quality
GMP Facilities
Lexington, MA : Primary manufacturing facility (100,000+ sq ft)Amsterdam, EU : Process development and analyticsCapacity : Multiple 2000L bioreactor trainsQuality Systems
FDA and EMA approved manufacturing
Full GMP compliance
Integrated quality management
Financial Overview
Exchange : NASDAQSymbol : QUREIPO : 2014Revenue
Hemgenix Sales : Commercial revenues from Hemophilia B therapyPartnerships : Strategic collaborations with major pharmaceutical companiesPartnerships
Major Collaborations
CSL Behring : Hemophilia B partnership (Hemgenix commercialization)Bristol-Myers Squibb : CNS research collaboration (historical)Genentech : Huntington's disease collaboration (historical)Research Publications uniQure maintains an active publication record:
Molecular Therapy publications on Huntington's program
Blood publications on hemophilia programs
Multiple conference presentations
[Huntington's Disease](/diseases/huntingtons-disease)
[Alzheimer's Disease](/diseases/alzheimers-disease)
[Parkinson's Disease](/genes/ar)
[Gene Therapy](/therapeutics/gene-therapy)
[AAV Vectors](/therapeutics/aav-vectors-neurodegenerative-gene-therapy)
[Huntingtin Protein](/proteins/huntingtin-protein)
External Links
[uniQure Official Website](https://www.uniqure.com/)
[NASDAQ: QURE](https://www.nasdaq.com/market-activity/stocks/quire)
[Hemgenix Website](https://www.hemgenix.com/)
[Clinical Trials](https://clinicaltrials.gov/)
References [@clinical]: [Clinical Trials - AMT-130](https://clinicaltrials.go[^5]: [Molecular Therapy Publications](https://www.cell.com/moleculartherapy/home)
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