Source: Philadelphia Business Journal by John George | March 3, 2020

Amicus Therapeutics will officially unveil its $25 million global research and gene therapy center in Philadelphia on Thursday.

The New Jersey biotechnology company last year entered into a 10-year lease deal under which it will occupy the top three floors at, and become the anchor tenant of, 3675 Market Street in University City. The 14-story building, which opened in November 2018, was developed by the University City Science Center, Wexford Science+Technology, and Ventas.

Amicus (NASDAQ: FOLD) is occupying about 75,000 square feet of space at the high-rise.

“Our global research and gene therapy center occupies the 13th and 14th floor,” said John Crowley, CEO of Amicus. “That’s where all the great ideas will turn into great science.”

Crowley said the labs feature an open-space design. “You can stand on one side and look through the glass walls and see out the other side,” he said.

Another feature will be portraits of patients with rare diseases for whom the company is developing treatments.

The costs to create the company’s lab space and offices are being shared by Amicus and the building’s owners, Crowley said.

Amicus has already hired about 100 people, mostly research scientists, to staff the center. Crowley expects that number to grow to about 200 over the next several years. He said the company plans to complete work this year on its offices on the 12th floor, which will house support staff — including program management and finance personnel — for the research team.

Amicus has another 600 employees at its headquarters in Cranbury, New Jersey, and in the 27 countries around the world where the company has a presence.

Last year, after Amicus first disclosed its plans to set up the research and gene therapy center in Philadelphia, Crowley told the Business Journal his company looked at all the major biotech hubs and academic centers before deciding on a location.

“We chose Philadelphia because we found it to be the most exciting and vibrant of all the hubs in the country,” Crowley said. “There’s a great coordination taking place between the industry and the academic centers and the hospitals and the city and state. That’s rare.”

In an interview Monday, Crowley said he has started referring to the region as the “cradle of cures” — a play off the historical references to Philadelphia, where both the Declaration of Independence and the U.S. Constitution were signed, as the Cradle of Liberty — because of the region’s history of medical advancements including cell and gene therapy.

The location of the company’s gene therapy center in uCity Square is near its major gene therapy partner: the University of Pennsylvania. Crowley said the collaboration was forged to combine his company’s expertise in protein design and engineering with Penn’s expertise in gene therapy to create new treatments for a variety of rare diseases.

Dr. James Wilson, who heads the gene therapy program at Penn, will be among the speakers at Thursday’s event to mark the opening of the center.

In May, Amicus and Penn’s Perelman School of Medicine agreed to a major expansion of their gene therapy research collaboration. Financial terms of the expanded research and licensing agreement include Amicus making a $10 million annual investment in Penn’s gene therapy program for five years in return for exclusive, disease-specific product development rights.

Founded in 2002, Amicus has one approved product, Galafold, used to treat Fabry disease, a progressive genetic disorder causing an enzyme deficiency that impairs kidney function, and another in late-stage development to treat Pompe disease, an inherited lysosomal disorder.

Crowley said its gene therapy program has grown to more than 50 programs targeting rare diseases such as Batten disease, a broad class of rare, fatal, inherited disorders of the nervous system.

Amicus posted revenues of $182.2 million in 2019, up from $91.2 million the previous year. Its net loss was $356.4 million last year, slightly larger than its $349 million net loss the previous year. The company ended 2019 with cash, cash equivalents and marketable securities totaling $452.7 million.