European gene-therapy pioneer to expand manufacturing with U.S. plant

UniQure has scored capital and talent to build out 55,000 square feet of manufacturing space for production of gene therapies. The planned Lexington, MA, facility is expected to complement existing manufacturing capacity at its home base in Amsterdam.

The biotech company, which makes the first approved gene therapy in the West, has reeled in $10 million in venture debt from Hercules Technology Growth Capital to support construction at the Lexington site. Lance Weed, who previously oversaw Amgen's ($AMGN) construction of a drug-production facility in Woburn, MA, will start work on Sept. 1 as uniQure's vice president of U.S. operations.

UniQure's Lexington facility is expected to manufacture therapies that use adeno-associated virus (AAV) gene therapies, which deliver healthy genes into cells to treat diseases. In November 2012, the company won European Union approval for Glybera, a gene therapy for the extremely rare disease lipoprotein lipase deficiency (LPLD). The company does not have FDA approval of Glybera, but last month uniQure CEO Jörn Aldag said his company would clarify its plans for getting the therapy approved in the U.S. in the next 12 to 18 months.

uniQure CEO Jörn Aldag

The company aims to use the added manufacturing capacity to become a major player in gene therapy, and the Lexington facility would help establish the group as a go-to partner in the field, able to produce plenty of supplies for clinical trials and commercialization. In addition to gene therapy for LPLD, the biotech group wants to use the approach to develop treatments for hemophilia, hearing loss, Parkinson's disease and Sanfilippo syndrome.

"The construction of this facility represents our commitment to establishing a global, fully integrated gene therapy company as we continue to build on our leadership position in the field of AAV-based gene therapy products," Aldag said in a statement.

Plans call for the Lexington facility to become operational in early 2015.

- here's the release