Manufacturing

State-of-the-art gene therapy manufacturing capability

uniQure is committed to manufacturing excellence in gene therapy, and we believe that our leading manufacturing capabilities provide us a significant competitive advantage in rapidly delivering gene therapies to patients. 

We produce AAV-based gene therapies in our own facilities with our proprietary manufacturing process, which uses insect cells and baculoviruses, a common family of harmless viruses found in invertebrates. uniQure is committed to manufacturing excellence in gene therapy. We believe that our leading manufacturing capabilities provide us a significant competitive advantage in rapidly delivering gene therapies to patients.  And with our high-speed and larger-scale manufacturing competencies for sizeable indications beyond orphan diseases, we aspire to lead the disruptive technology field in this area.

Modular Platform
Our facility in Lexington, Massachusetts, is one of the largest, most versatile gene therapy manufacturing facilities in the world. uniQure has made significant investments in designing, constructing and equipping our facility with state-of-the-art laboratories and commercial-scale production capabilities. The facility, which was expanded in late 2019 from 55,000 square feet to 80,000 square feet, produces our AAV-based gene therapies with a proprietary, commercial-scale, current good manufacturing practices (“cGMP”)-compliant, manufacturing process. It provides us production capabilities to support all of our existing programs, including clinical as well as commercial material, with flexibility to expand further. 

We develop AAV production process with the commercial requirements in mind to avoid any CMC issues during the development of our product. 

Increasing Capacity for Future Growth
We recently completed the expansion of our Amsterdam site to build additional laboratories to support new research and development activities, as well as the construction of a cleanroom designed to be capable of manufacturing cGMP materials at a 500-liter scale. This gives us similar capabilities in our two sites, which allows us to be quicker and more agile in product development. We are committed to continuing to improve our cost effectiveness to make gene therapy always more affordable to patients with unmet medical needs. 

Gene therapy manufacturing sidebar