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Philadelphia’s biotech research power lures gene therapy company Amicus Therapeutics to city


By David Schwartz
Published: March 12th, 2019

Amicus Therapeutics, a growing biopharmaceutical company that focuses on rare and orphan diseases, will launch a new Global Research and Gene Therapy Center at uCitySquare in Philadelphia, citing the city as a burgeoning hub for medical breakthroughs as a primary factor in the move.

When the company’s 75,000-square-foot center opens in late 2019, it will house 200 new hires — biologists and chemists – along with the firm’s gene therapy leadership team. Amicus will retain its corporate headquarters in Cranbury, NJ.

“It’s a big deal for us, and I hope for Philadelphia, as well,” said CEO John F. Crowley. Amicus will collaborate with top gene therapy researchers at the University of Pennsylvania and Penn Medicine, and seek additional partnerships with Drexel University and other regional institutions, he said. “For us Philadelphia is a terrific fit to make medicines for people who have rare disorders.”

Crowley’s dramatic and unusual history in biotech was retold in the 2010 movie Extraordinary Measures, starring Brandon Frasier and Harrison Ford.

As a newly minted Harvard MBA, Crowley’s two youngest children were diagnosed as infants with Pompe disease, a rare ailment that ravages muscles and nerve cells. Rather than accept a diagnosis that would have consigned his kids to death at 8 years of age, Crowley  launched a company that pioneered enzyme replacement therapy and saved his children’s lives.

“It wasn’t a cure, but it improved their muscle strength and kept them alive,” he said. The company on Monday won breakthrough therapy designation from the FDA for its treatment for a late-manifesting form of the disease.

Crowley likens University City’s energy to that of the famed Kendall Square neighborhood of Cambridge, MA, which has parlayed research from MIT, Harvard, and other universities into a powerful biotech ecosystem. “This very much reminds me of what Kendall Square was a few years ago,” Crowley said. “This is a terrific environment and we’re just thrilled.”

Christopher P. Molineaux, president and CEO of Life Sciences Pennsylvania, called the Amicus announcement a significant development in the life sciences community of Philadelphia. “They will join the scientific and business prowess of our world-leading ecosystem, while bringing considerable jobs, expertise and innovation to tackle unmet medical needs for the ultimate beneficiary: patients.”

Crowley said proximity to the University of Pennsylvania and professor Jim Wilson of Penn Medicine’s Orphan Disease Center was a major draw. “We formalized a major collaboration with Dr. Wilson in the fall of 2018, and now we’ll be literally down the street from him.”

Amicus will be investigating gene therapy cures for Pompe disease and 14 other lethal diseases.

“This is not CRISPR,” Crowley said, referring to the novel gene editing technology. “This is gene therapy, not gene editing. Dr. Wilson at Penn has been at the center of gene therapy. If this works, this should be a onetime treatment.”

Source: The Inquirer

Posted under: University-Industry Engagement Week

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