UniQure N.V. has announced that is has “scaled up” the manufacturing process at its large facility in Lexington, MA. According to a uniQure press release, the facility is designed to manufacture the company’s proprietary hemophilia B gene therapy candidate, AMT-060.
AMT-060, which has been developed to treat patients with hemophilia B (factor IX deficiency), is manufactured with UniQure’s adeno-associated viruses (AAV). These small viruses, which do not cause disease and typically produce mild immune responses, are used as vectors (delivery vehicles) to introduce a functioning factor IX (FIX) gene into the liver cells of patients with hemophilia B. The goal of the trial is to trigger long-term FIX protein production through a single administration of the therapy. This could dramatically reduce the frequency of bleeding episodes in people with hemophilia B.
“We have now achieved the successful manufacturing of AMT-060 at our Lexington facility at a scale sufficient to support pivotal clinical trials and commercial supply,” stated Matt Kapusta, chief executive officer of uniQure. “We have made significant progress over the past several months to now be in a position where we have developed a commercial-scale process and are evaluating our completed batches to assess comparability. We look forward to finalizing this work in anticipation of meetings with regulators to further discuss plans to advance our hemophilia B program into a pivotal study next year.”
UniQure expects to meet with the U.S. Food and Drug Administration and European Medicines Agency in the early fall.
Source: uniQure press release dated July 21, 2017
