On March 5, 2020, Amicus Therapeutics announced the official opening of its new Global Research and Gene Therapy Center of Excellence in Philadelphia, PA, to advance its portfolio of rare disease gene therapy programs. Amicus and the University of Pennsylvania (Penn) had earlier announced a major expansion to their gene therapy collaboration in 2019, which provides Amicus with disease-specific worldwide rights to Penn’s next-generation gene therapy technologies from Wilson Lab. The gene therapy technologies focus primarily on lysosomal storage disorders as well as twelve additional more prevalent rare diseases, including Rett Syndrome, Angelman Syndrome, and select other muscular dystrophies.

The 75,000-ft2 center is located on the top three floors of the new building and consists of office and state-of-the-art laboratories. It will ultimately house approximately 200 researchers and drug developers focused exclusively on gene therapies.

“This is a remarkable advancement in the history of Amicus and further strengthens our great collaboration with Dr. Jim Wilson and the Gene Therapy Center at Penn. Philadelphia is a magnet for talent in gene therapy and an engine for innovation. This new global research center located in the ‘cradle of liberty’ will become part of the

‘cradle of cures’ as we move many gene therapy programs forward toward patients in need. With exclusive global rights to 50 rare diseases in collaboration with Dr. Wilson’s team we hope to be able to alleviate an enormous amount of human suffering with the great science work that will be done in this new facility,” stated John F. Crowley, chairman and CEO of Amicus, in a company press release.