Philadelphia, which is developing its reputation as a hotbed of biotechnology research and development, is poised to play host next week to the world’s largest biotech industry convention, the BIO International Convention.
As many as 20,000 people from 75 countries are expected to flock to the four-day convention at the Pennsylvania Convention Center in Center City. Among them will be superstars of gene therapy, finance, and cancer research.
Keynote speakers will include Jamie Dimon, CEO of JPMorgan Chase, and Siddhartha Mukherjee, a Columbia University cancer physician and Pulitzer Prize-winning author of The Emperor of All Maladies.
The three governors from the tri-state area -- Tom Wolf of Pennsylvania, Phil Murphy of New Jersey, and John Carney of Delaware -- will hold what’s billed as "a conversation” on Tuesday about the impact of the life sciences industry in their states, the nation’s opioid crisis, and how they are grappling with the rising cost of health care.
“It’s not a trade show,” said Jim Greenwood, the former Bucks County congressman who is president of the Biotechnology Innovation Organization (BIO), which stages the annual convention. “It’s a celebration of biotech, one of the most profound things we as humans have ever done. It’s all about understanding how life works on the cellular, genetic, and molecular level.”
BIO represents and lobbies on behalf of more than 1,100 biotechnology companies, academic institutions, state biotech centers, and related groups across the U.S. and internationally, Greenwood said.
Greenwood cheekily refers to Greater Philadelphia as “Cell-icon Valley,” a nod to the region’s leadership in the realm of cell and gene therapy. According to Greenwood’s group, the tri-state area contains the sixth largest biotech cluster in the country and the city sits alongside Boston and San Francisco as having one of the world’s most dynamic life science communities.
This is the fourth time that Philadelphia has landed the global BIO convention, where business developers for biotech companies hobnob with researchers, entrepreneurs, pharmaceutical executives, and specialists in regulatory affairs.
“It provides a relaxed forum for people from wide sectors of the industry to meet and see if there are ways to work together,” said Katherine High, president and head of R&D at Spark Therapeutics, the West Philadelphia gene therapy powerhouse that was acquired this year by Roche for $4.8 billion. The acquisition is set to close later this year.
At Spark, High led the development of the first gene therapy to win FDA approval for a genetic disease. Spark’s Luxturna is a once-and-done treatment for a rare form of blindness. Though pricey at $850,000, Luxturna has a 93 percent rate of success, she said.
High recognizes that the cost can be hard to swallow.
“The problem is that the whole drug system is based on giving a medicine chronically," she said. “Gene therapy offers the promise of a one-time treatment. How do you reward a single treatment over something that has to be given repetitively?”
At BIO, High is scheduled to appear on a panel about how to realize the promise of gene therapies for patients around the world.
“There’s tremendous activity in this space,” High said. “And it’s accelerating.”
In the last two years, the number of investigational new drug applications involving gene therapy -- the first step in gaining FDA approval -- has doubled to more than 800, she said.
Hemophilia, Huntington’s Disease, and the blood disorder Thalassemia are among the prime targets of gene therapy research, she said. “But I think the prize [for potential treatments] is sickle cell disease” which affects 4.4 million people.
Spark has ongoing studies focusing on hemophilia A and a treatment for hemophilia B is in Phase 3 trials. High said Spark is working to develop a program to treat Huntington’s, but it’s still in the pre-clinical stages.
Eventually, she expects research will deliver therapy or even cures for disorders caused by more than one gene. Diseases linked to flaws in multiple genes include cancer, congestive heart failure, and Alzheimer’s.
“Already, there are ongoing trials for age-related macular degeneration and congestive heart failure, but they didn’t work,” she said. “For a lot of things in therapeutics, a failed trial paves the way for a trial that works.”
Other highlights of the convention include: