UniQure
Developer of AAV-based gene therapies and commercial-grade AAV manufacturing. Focused on liver-directed and central nervous system indications, the company operates a modular gene therapy platform, conducts multi-phase clinical programs (including CNS surgical delivery), and provides in-house GMP manufacturing and CMC capabilities to advance rare-disease and neurological therapeutics.
Industries
Nr. of Employees
medium (51-250)
Products
AAV-delivered gene therapy for hemophilia B (commercially approved)
Commercially available AAV-based gene therapy for hemophilia B developed using the company's AAV platform and manufactured at its commercial-grade facility.
Clinical-stage AAV gene therapy candidates (CNS and metabolic indications)
Investigational AAV-based programs in development for Huntington’s disease, temporal lobe epilepsy, Fabry disease, SOD1-ALS and other rare indications.
HEMGENIX
HEMGENIX (etranacogene dezaparvovec) is a one-time AAV5-based gene therapy for hemophilia B that enables patients to produce their own factor IX.
AMT-061
An investigational AAV5-based gene therapy utilizing the FIX Padua transgene for the treatment of hemophilia B.
AAV delivery system
Best-in-class AAV delivery system focused primarily on liver-directed and central nervous system disorders.
Glybera
Alipogene tiparvovec, the first approved gene therapy in the Western world, indicated for lipoprotein lipase deficiency.
AAV-delivered gene therapy for hemophilia B (commercially approved)
Commercially available AAV-based gene therapy for hemophilia B developed using the company's AAV platform and manufactured at its commercial-grade facility.
Clinical-stage AAV gene therapy candidates (CNS and metabolic indications)
Investigational AAV-based programs in development for Huntington’s disease, temporal lobe epilepsy, Fabry disease, SOD1-ALS and other rare indications.
HEMGENIX
HEMGENIX (etranacogene dezaparvovec) is a one-time AAV5-based gene therapy for hemophilia B that enables patients to produce their own factor IX.
AMT-061
An investigational AAV5-based gene therapy utilizing the FIX Padua transgene for the treatment of hemophilia B.
AAV delivery system
Best-in-class AAV delivery system focused primarily on liver-directed and central nervous system disorders.
Glybera
Alipogene tiparvovec, the first approved gene therapy in the Western world, indicated for lipoprotein lipase deficiency.
Services
Partnering with external research organizations to identify and evolve AAV variants optimized for liver and CNS delivery.
Clinical and commercial manufacturing of AAV vectors using an insect-cell/baculovirus platform combined with CMC and analytical development support.
End-to-end clinical trial execution for multi-phase studies including site network management, surgical delivery coordination (MRI-guided CED) and participant support.
Commercial forecasting, launch analytics, and KPI reporting using BI and cloud analytics tools to support business strategy and product launches.
Partnering with external research organizations to identify and evolve AAV variants optimized for liver and CNS delivery.
Clinical and commercial manufacturing of AAV vectors using an insect-cell/baculovirus platform combined with CMC and analytical development support.
End-to-end clinical trial execution for multi-phase studies including site network management, surgical delivery coordination (MRI-guided CED) and participant support.
Commercial forecasting, launch analytics, and KPI reporting using BI and cloud analytics tools to support business strategy and product launches.
Expertise Areas
- Gene therapy development
- AAV vector engineering
- CMC and GMP AAV manufacturing
- Clinical trial management (neurology and rare diseases)
Key Technologies
- AAV vectors and serotype engineering
- Baculovirus–insect cell AAV manufacturing
- Gene cassette design (tissue-specific promoters, microRNA scaffolds)
- MRI-guided convection-enhanced delivery (CED)
News & Updates
Webcast presentation for pivotal HD program.
Archived earnings calls, conference presentations and program updates covering clinical programs and corporate results.
Company highlights being behind the first approved gene therapy in the western world and subsequent commercial manufacturing achievements.
Construction and operation of a commercially licensed gene therapy manufacturing facility (Lexington, MA).
Webcast presentation for pivotal HD program.
Archived earnings calls, conference presentations and program updates covering clinical programs and corporate results.
Company highlights being behind the first approved gene therapy in the western world and subsequent commercial manufacturing achievements.
Construction and operation of a commercially licensed gene therapy manufacturing facility (Lexington, MA).