Amicus Opens New Global Research and Gene Therapy Center of Excellence

Rare Daily Staff

Amicus Therapeutics said it has opened its Global Research and Gene Therapy Center of Excellence in Philadelphia to advance its portfolio of rare disease gene therapy programs.

In 2019, Amicus and the University of Pennsylvania announced an expansion of their gene therapy collaboration, which provides Amicus with disease-specific worldwide rights to Penn’s next generation gene therapy technologies from the Wilson Lab for most lysosomal storage disorders, as well as 12 more prevalent rare diseases including Rett syndrome, Angelman syndrome and select other muscular dystrophies.

The 75,000 square foot center will ultimately house about 200 researchers and drug developers focused exclusively on gene therapies.

“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,” said John Crowley, chairman and CEO of Amicus Therapeutics. “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.”

Photo: John Crowley, chairman and CEO of Amicus Therapeutics