Amicus Opens Gene Therapy Center Focused on Rett Syndrome, Other Rare Diseases

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by Vanessa Pataia |

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Amicus announcement

Kate Stringaris, a researcher with the NIH’s National Heart, Lung and Blood Institute in Bethesda, Maryland, separates lymphocytes from a blood sample as part of an investigation into natural killer immune cells.

Amicus Therapeutics has opened its Global Research and Gene Therapy Center of Excellence that will seek to advance research programs in Rett Syndrome and several other rare diseases.

Besides Rett, the Center will focus on Angelman Syndromemuscular dystrophies and lysosomal storage disorders, such as Pompe and Fabry diseases. Collectively, lysosomal storage disorders result from a lack of certain enzymes within lysosomes — cellular structures in which large molecules are broken down. People with one of these disorders have a toxic buildup of such molecules in their cells.

The Center is located on the top three floors of a new building at 3675 Market St. in uCity Square, Philadelphia. The building has offices and state-of-the-art laboratories, with plans to house approximately 200 researchers and  developers working solely 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,” John F. Crowley, chairman and CEO of Amicus, said in a press release.

In 2018, Amicus established a research collaboration and license agreement with the University of Pennsylvania (UPenn) to develop programs in four rare genetic diseases, including Pompe and Fabry diseases. The agreement provided funding to advance preclinical research at the laboratory of James M. Wilson, MD, PhD, at UPenn. The pact also covered funding to license newly developed technologies.

In May 2019, Amicus and UPenn announced an expansion to six rare genetic disease programs, including Sanfilippo syndrome. The new agreement also gave Amicus worldwide rights to Penn’s next-generation gene therapy technologies for most lysosomal storage disorders and 12 more rare diseases, including Rett, Angelman syndrome and certain muscular dystrophies.

Opening the new center “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,” Crowley said.

“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,” Crowley said.

A ribbon-cutting event celebrated the opening of the new center on March 5, with special guests that included Dr. Wilson, government officials and people with rare diseases and their families.