Amicus Research and Gene Therapy Center To Focus on Batten, Other Diseases

To advance its commitment to research aimed at improving the lives of those with rare metabolic disorders such as Batten disease, Amicus Therapeutics is establishing a Global Research and Gene Therapy Center of Excellence in Philadelphia, Pennsylvania.

The 75,000-square-foot center, scheduled to open this year, will be in uCity Square, a 6.5 million-square-foot, mixed-use community consisting of retail, residential, clinical, office, and laboratory space, concentrated around a public square, surrounded by academic centers and hospitals.

Philadelphia is a hub for biotechnology and gene therapy research. Amicus views the city as the optimal place to collaborate, promote its therapy pipeline, and attract and retain top scientists, the company stated in a news release.

The center is an “important next step in the evolution of our science, research, and gene therapy capabilities,” John F. Crowley, Amicus’ chairman and chief executive officer, said in the statement. “In considering locations, Philadelphia became the clear choice as a burgeoning hub for medical breakthroughs.”

Crowley cited the center’s proximity to collaborators at the University of Pennsylvania and other institutions, as well as to the biotechnology company’s Cranbury, New Jersey, headquarters. The Philadelphia site will house the global science organization and gene therapy leadership team. Amicus plans to hire 200 employees, including biologists and chemists.

”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,” Crowley said.

The Center of Excellence will be led by Jeff Castelli, chief portfolio officer and newly appointed head of gene therapy, and Hung Do, chief science officer. For now, about a dozen scientists have moved into a temporary space in West Philadelphia.

”The City of Philadelphia is committed to fostering innovative companies, academic institutions, and hospitals that are focused on the latest advancements in research and development, while also elevating the patient experience within our healthcare systems,” Philadelphia Mayor Jim Kenney said in the statement.

“Amicus Therapeutics is an established leader in biotechnology with a unique and intense patient-dedicated mission. The company’s presence and investment in Philadelphia will create additional opportunities that will be highly influential as our city continues its transformation into a major global biotech hub,” he said.

Amicus is currently recruiting children for its Phase 1/2 clinical trial (NCT03770572), which will evaluate the safety and effectiveness of a gene therapy, identified as AAV9-CLN3, for children ages 3–10 who have a confirmed diagnosis of CLN3 Batten disease.