Amicus Therapeutics, a biopharmaceutical company focused on rare metabolic diseases, is opening a new Global Research and Gene Therapy Center of Excellence in Philadelphia next to the University of Pennsylvania campus.

“This is an important next step in the evolution of our science, research, and gene therapy capabilities,” said John Crowley, chairman and CEO of Amicus.

In considering locations, Crowley said Philadelphia became the clear choice as a burgeoning hub for medical breakthroughs with proximity to Amicus collaborators at the University of Pennsylvania, other major academic centers, and hospitals in the area.

The establishment of a new R&D center comes just months after Amicus signed two broad agreements in gene therapy. In September 2018 the biotech acquired worldwide development and commercialization rights for ten gene therapy programs developed at The Center for Gene Therapy at The research Institute at Nationwide Children’s Hospital and Ohio State University. One month later, Amicus entered into a broad research and collaboration agreement with the Gene Therapy Program at Penn to develop AAV gene therapies for four genetic disorders, including Pompe disease, Fabry disease, CDKL5, and an undisclosed rare metabolic disorder.

“Amicus has developed unique abilities in drug development in the lysosomal storage disorders, particularly in Pompe and Fabry diseases,” said James Wilson, head of Penn’s Orphan Disease Center at that time. “There are multiple and unique challenges in developing optimal gene therapy products for patients living with Pompe and Fabry diseases. I believe that we can combine the technologies and capabilities from my research laboratory at Penn with the Amicus expertise in protein engineering, glycobiology and disease biology understanding to rapidly advance novel gene therapies to the clinic.”

The new facility will be located in uCity Square, a 6.5 million square-foot, mixed-use knowledge community consisting of office, laboratory, clinical, residential and retail space designed to enable university and corporate research, entrepreneurial activity and community engagement. The new 75,000 square foot space will be completed in the second half of 2019 and will house about 200 employees, serving as the headquarters of Amicus’ science organization and gene therapy research team. An initial group of Amicus researchers have already moved into temporary space nearby.

“As Amicus continues to expand globally, my hope is that the great science to come from our research in Philadelphia will one day soon lead to medicines with the potential to alleviate an enormous amount of suffering.,” said Crowley. “As Amicus continues to expand globally, my hope is that the great science to come from our research in Philadelphia will one day soon lead to medicines with the potential to alleviate an enormous amount of suffering. This is our mission at Amicus and we are honored to be a part of the exciting Philadelphia research community.”

Photo: John Crowley, chairman and CEO of Amicus