Cradle of Cures – Site Selection Magazine Feature

This article originally appeared in Site Selection Magazine’s July 2020 edition.

Greater Philadelphia is a center of excellence in cell and gene therapies.

Long revered as America’s “cradle of liberty,” Philadelphia is leading another revolution. Cell and gene therapies being created in the city’s prodigious cluster of clinical labs offer hope for treating the untreatable and curing the incurable. It is a young and fast-emerging field of modern medicine, and its cutting edge is Philadelphia.

“Philadelphia is becoming the ‘cradle of cures,’ a beacon for this entire industry,” says Bradley Campbell, president and COO of New Jersey-based Amicus Therapeutics. Amicus, having scoured medical hubs in the U.S. and Europe, chose Philadelphia as the site of its Global Research and Gene Therapy Center of Excellence, which opened
in March.

“We wanted to have a state-of-the-art lab and office facility that could help us attract the best gene therapy scientists in the world as we look to discover and develop next generation therapies for patients with rare diseases,” Campbell tells Site Selection. “We think Philadelphia is the perfect place for that.”

It’s no coincidence that Amicus built its $25 million facility in West Philadelphia’s University City, home to the University of Pennsylvania School of Medicine, with which it has a partnership, and to Childrens Hospital of Philadelphia (CHOP). UPenn, working with Novartis, made medical history in 2017 when its leukemia treatment
for children and young adults became the first cell therapy to be approved by the FDA.

Shortly thereafter came approval for the nation’s first gene therapy, also developed in Philadelphia. Luxterna, a one-time treatment for inherited blindness, emerged from a collaboration between CHOP and Philadelphia’s Spark Therapeutics. Now developing a gene therapy treatment for hemophilia, Spark recently was acquired by Switzerland’s Roche for $4.3 billion.

Such signature achievements have helped to serve as a springboard. Philadelphia now is home to a growing roster of more than 30 cell and gene therapy development companies. Many in the region and in the industry have adopted the moniker “Cellicon Valley” to describe the region’s closely-knit biotech community. It’s a fitting description.

“There’s truly an incredible proportion of the world’s cell and gene therapy companies in University City and other places in the Philadelphia region,” says Joan Lau, CEO of Spirovant Sciences, another of Philadelphia’s emerging gene therapy companies. “What is happening here is extraordinary, and it can only occur with the type of density we have.”

A Strong Foundation for Growth

Philadelphia’s cell and gene therapy cluster has absorbed investment of more than $1.7 billion since the second half of 2015, according to the Cell & Gene Therapy and Connected Health Initiative led by the CEO Council for Growth. That is but one of many indicators of the perceived potential of advanced immunotherapies.

“What’s beautiful about gene therapy is that it’s extraordinarily targeted,” Spirovant’s Lau tells Site Selection. “You’re not looking at taking a pill and hoping it goes to the right place. You’re designing the molecule to target precisely the tissue or the organ that you are trying to alter.”

That only about 20 such therapies have received approval to date testifies to the ardor, expertise and investment required to shepherd potential treatments from concept to reality. Ideas come to fruition along a complex chain whose links include academia, hospitals, testing labs, contract researchers and contract manufacturers.

Philadelphia-based Imvax, whose lead program is a treatment for an aggressive form of brain cancer, sprang from research developed at Thomas Jefferson University. Chief Financial Officer Art Howe says Philadelphia has a deeply developed infrastructure that conveys distinct advantages upon its gene therapy cluster.

“There’s an incredibly rich ecosystem of suppliers and service providers,” Howe tells Site Selection. “There are hundreds and hundreds of companies that are part of this ecosystem, and that’s hugely important.”

Shanghai-based Wuxi AppTech is an increasingly prominent player in that shared ecosystem. Wuxi is the largest tenant at the South Philadelphia Navy Yard, a former military base that complements University City as a thriving hub of next generation medicine. Felix Hsu, Chief Commercial and Business Officer, describes Wuxi as “an enabler of innovation” for advanced therapy enterprises large and small.

“We help them develop their products, and then we help them manufacture those products,” Hsu says. Wuxi is nearing completion of its fourth building on the Navy Yard campus, a 95,000 sq.-ft. (8,900 sq.-m.) office and laboratory. Its clients include California-based Iovance Biotherapeutics, a cancer therapy firm, now moving into a sprawling manufacturing space at the Navy Yard.

Talent Leads the Way

As rapidly evolving technologies, cell and gene therapies crave talent. They find it in Philadelphia, home not only to UPenn, CHOP and Jefferson, but to other highly-regarded academic institutions such as Temple University, Drexel University, the University of the Sciences and the Wistar Institute, a nonprofit research center for
biomedical science.

“Everything we do stops and starts with people, and Philadelphia is a hotbed for developing talent in the scientific and disease areas,” says Usman “Oz” Azam, president and CEO of Philadelphia based Tmunity, which is developing immunotherapies for cancers and autoimmune diseases. Drawn by an attractive lifestyle and a cost of a living distinctly lower than the biotech hubs of Boston and San Francisco, Philadelphia is drawing talent from elsewhere, as well. Azam speaks of a “positive feedback loop” taking hold.

“Attracting more firms provides more job opportunities and thus attracts more talent from outside the Philadelphia region, which in turn makes Philadelphia even more desirable to relocating firms,” he says.

The cell and gene therapy space is poised for a breakout. The FDA expects a doubling of applications for treatments this year, and analysts predict annual approvals by mid-decade equivalent to the total number certified up to now. Imvax’s Howe believes Philadelphia is poised to reap the fruits of what it has sewn.

“Local leaders here have been tremendously supportive and proactive. They recognize the impact of this vibrant life sciences community. They are striving mightily to get additional funding for companies to locate here. They understand,” he says, “that this is a large part of the future of this city.”

This investment profile was prepared under the auspices of Select Greater Philadelphia. For more information, contact Meghan Kelly at 1-215-790-3726 or by e-mail at [email protected] Select Greater Philadelphia also presents a podcast showcasing stories of business successes and economic development across the Greater Philadelphia region.

Philly Biotech Is On It

From our region’s epicenter of discovery, Greater Philadelphia researchers and companies are part of the global race to get a handle on COVID-19, including Biomeme, The Wistar Institute, and the newly formed Penn Center for Research on Coronaviruses and Other Emerging Pathogens (PENNCoV).

Article from The Philadelphia Citizen

Amicus opens UCity R&D tower, calls Philly the ‘Cradle of Cures’

The future of research and development, in rare diseases and beyond, is Philadelphia.

By moving its R&D team to University City this year, publically traded gene therapy company Amicus Therapeutics now calls the Greater Philadelphia region’s bustling cell and gene therapy ecosystem home.

Article from the Philadelphia Inquirer

Biotech company with U.S. headquarters in Montco raises $130 million in series B

The biotech company Immunocore, with its U.S. headquarters in Montgomery County, announced a $130 million Series B financing round to continue the development of T-cell receptor therapies.

Article from the Philadelphia Business Journal

Penn gene therapy spinout exceeds projections with $216M IPO

Penn Medicine spinout Passage Bio raised $216 million through an initial public offering to support the continued development of clinical trials for gene therapies to treat rare central nervous system disorders – exceeding expectations and proving that cell and gene therapies can thrive in the Greater Philadelphia region.

Article from the Philadelphia Business Journal

This isn’t a ‘biotech bubble,’ it’s ‘Philadelphia’s moment’ to become a center for gene therapy, says Penn-based CEO

“This is not a bubble, it’s Philadelphia’s moment,” Steven Nichtberger, CEO of Cabaletta Bio offers his perspective on the region’s exponential growth as the epicenter of cell and gene therapy.

Article from the Philadelphia Inquirer

PACT 2019 Philadelphia Venture Report

PACT Philadelphia’s annual report ranks the region seventh in the country for VC funding after a record-setting $2.5 billion invested in 2019, fueled by a steady stream of life sciences deals and activity among cell and gene therapy and connected health companies.

Report from PACT

CRISPR’d Cells Show Promise in First U.S. Human Safety Trial

Last year, three cancer patients were treated with their own CRISPR-edited T cells as part of a groundbreaking U.S. trial to determine the safety of such a treatment.

The result? A promising breakthrough for gene editing.

Article from Wired

Biotech Tour: Inside the Newly Rehabbed Spring House Innovation Park

See inside the new Spring House Innovation Park, a soon-to-be hub of life sciences innovation housing companies like Anpac Bio – which will open its U.S. HQ and very first east coast clinical lab site on the campus.

Article from Philadelphia magazine

Philadelphia gene therapy startup plans $125M IPO

Another life sciences IPO for Greater Philadelphia: Gene therapy startup Passage Bio plans to go public via a $125 million IPO, to continue developing six gene therapy candidates for central nervous system diseases, including dementia.

Article from the Philadelphia Business Journal