UniQure Biopharma B.V.
uniQure is dedicated to reimagining the future of medicine by delivering innovative cures that transform lives through genomic medicine and gene therapy. With over 20 years of industry experience, the company focuses on developing transformative gene therapies for serious diseases, fostering a collaborative culture, and maintaining a strong commitment to patients, innovation, and social responsibility.
Industries
Nr. of Employees
large (251-1000)
UniQure Biopharma B.V.
Products
AAV-based gene therapy program for SOD1-associated ALS
A CNS-targeted AAV-delivered microRNA therapeutic candidate designed to reduce SOD1 expression; supported by preclinical rodent and non-human primate data and currently in Phase I/II clinical development.
Gene therapy program for Huntington's disease
Clinical-stage gene therapy program targeting Huntington's disease with designation for expedited regulatory review.
Gene therapy candidate for refractory temporal lobe epilepsy
Preclinical-to-clinical gene therapy candidate for refractory mesial temporal lobe epilepsy (rTLE) under development with supporting preclinical data.
Gene therapy programs for Fabry disease and hemophilia B
Programs advancing gene replacement or modifier strategies for metabolic and coagulation disorders, including an approved hemophilia B therapy referenced in corporate materials.
AAV-based gene therapy program for SOD1-associated ALS
A CNS-targeted AAV-delivered microRNA therapeutic candidate designed to reduce SOD1 expression; supported by preclinical rodent and non-human primate data and currently in Phase I/II clinical development.
Gene therapy program for Huntington's disease
Clinical-stage gene therapy program targeting Huntington's disease with designation for expedited regulatory review.
Gene therapy candidate for refractory temporal lobe epilepsy
Preclinical-to-clinical gene therapy candidate for refractory mesial temporal lobe epilepsy (rTLE) under development with supporting preclinical data.
Gene therapy programs for Fabry disease and hemophilia B
Programs advancing gene replacement or modifier strategies for metabolic and coagulation disorders, including an approved hemophilia B therapy referenced in corporate materials.
Services
Clinical development and trial execution
Design and conduct of early-phase clinical trials to assess safety, tolerability and exploratory efficacy of gene therapy candidates.
AAV manufacturing and CMC development
End-to-end manufacturing development including process development, scale-up, and CMC program management for AAV-based products.
Preclinical translational studies
Conduct of preclinical efficacy and safety studies in rodent and non-human primate models to support IND-enabling and clinical development.
Regulatory strategy and submissions support
Preparation and management of regulatory interactions, submissions and designation requests with health authorities.
Quality systems and compliance
Implementation and maintenance of global quality management systems and readiness for regulatory inspections.
Clinical development and trial execution
Design and conduct of early-phase clinical trials to assess safety, tolerability and exploratory efficacy of gene therapy candidates.
AAV manufacturing and CMC development
End-to-end manufacturing development including process development, scale-up, and CMC program management for AAV-based products.
Preclinical translational studies
Conduct of preclinical efficacy and safety studies in rodent and non-human primate models to support IND-enabling and clinical development.
Regulatory strategy and submissions support
Preparation and management of regulatory interactions, submissions and designation requests with health authorities.
Quality systems and compliance
Implementation and maintenance of global quality management systems and readiness for regulatory inspections.
Expertise Areas
- Gene therapy development
- AAV-based therapeutic development
- Clinical trial management (early-stage)
- CMC and GMP manufacturing for biologics
Key Technologies
- AAV vectors
- microRNA-mediated gene silencing
- Viral vector delivery to CNS
- Rodent and non-human primate disease models