Amicus Therapeutics to Hire 200 Researchers at New Gene Therapy Center in Philadelphia

headshot of Amicus Therapeutics' CEO John Crowley

Amicus Therapeutics, headquartered in Cranbury, New Jersey, announced it is launching a Global Research and Gene Therapy Center of Excellence in Philadelphia.

The new center will be located in uCity Square at 3675 Market Street. uCity Square is a 6.5 million-square-foot, mixed-use “knowledge community” made up of office, laboratory, clinical, residential and retail space. Its goal is to allow university and corporate research, entrepreneurial activities and community engagement.

The company announced on Monday, Feb. 25 that the U.S. Food and Drug Administration (FDA) had granted its AT-GAA for treatment of late-onset Pompe disease Breakthrough Therapy Designation (BTD). AT-GAA is the first investigational product for Pompe disease, an inherited lysosomal storage disorder, to receive BTD. AT-GAA is a unique gene therapy made up of ATB200, a recombinant human acid alpha-glucosidase (rhGAA) enzyme that is co-administered with AT2221, a pharmacological chaperone.

So far, about a dozen Amicus researchers have moved into a temporary space in West Philadelphia for the Center of Excellence. The temp site is at BioLabs@CIC Philadelphia. The company plans to hire 200 new people, biologists and chemists when it opens its 75,000 square-foot location in late 2019.

The company will keep its corporate headquarters in Cranbury, New Jersey, but the Philadelphia site will host its global science organization and gene therapy leadership team.

“This Amicus Global Research and Gene Therapy Center of Excellence is an important next step in the evolution of our science, research and gene therapy capabilities,” stated John F. Crowley, Amicus’ chairman and chief executive officer. “In considering locations, Philadelphia became the clear choice as a burgeoning hub for medical breakthroughs.”

He added, “The proximity to our collaborators at the University of Pennsylvania and other major academic centers and hospitals in the area also provides a tremendous opportunity to advance our commitment to gene therapies. Philadelphia is easily accessible to New Jersey, which has been a strong contributor to our success and will remain the location of our global headquarters.”

The Center of Excellence will be headed by Jeff Castelli, chief portfolio officer and recently appointed Head of Gene Therapy, and Hung Do, chief science officer.

Jim Kenney, Mayor of Philadelphia, stated, “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. 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.”

Crowley and his research efforts were told in the 2010 film Extraordinary Measures, starring actors Brendan Fraser and Harrison Ford. Fraser portrayed Crowley and Ford portrayed Dr. Stonehill, who was a composite of several researchers depicted in Geeta Ananda’s book, “The Cure,” including Bill Canfield.

Crowley’s two youngest children were diagnosed with Pompe disease. At that time, there was no effective treatments and death would have been likely by the age of eight. Crowley was a financial consultant with an MBA from Harvard. He launched Amicus to develop a treatment for his children. It developed an enzyme replacement therapy that saved his children’s lives.

“It wasn’t a cure,” Crowley noted, “but it improved their muscle strength and kept them alive.”

Its AT-GAA has the potential to expand on that development. It is currently in a global Phase III clinical trial, PROPEL, in adults with late-onset Pompe disease, as well as a global Phase I/II trial.

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