March: A guided tour of ‘Cellicon Valley
By Chris Satullo and Michael Matza
Four people who sit in the middle of the regional ferment over gene and cell therapy gave the Sunday Breakfast Club a "Guided Tour of 'Cellicon Valley'" last week at the club's March program at the Science History Institute.
Joan Lao, CEO and co-founder of Spirovant, a gene therapy company in West Philadelphia, got the evening's biggest laugh when she volunteered to take a shot at explaining how cell therapy, as opposed to gene therapy, works:
"Imagine there are cells that are expressing cancer antigens. Those antigens have a shape. Let’s just say the antigen looks like a Dallas Cowboy star on the surface of those cells. And what we want to do via cell therapy is eliminate cells that look like the Dallas Cowboy star. So, you arm immune cells, usually T-cells, so when you put them back into the body, they can hunt around for the Dallas Cowboy star cells and kill 'em. They also hunt down cells with the Houston Astros star shape, so it's a comprehensive system."
Laughter and applause followed.
Tiffany Wilson, CEO of the University City Science Center, also drew chuckles when she pointed out a contrast between the biotech community in Atlanta, where she used to work, and Philadelphia's, "The capacity here is absolutely as robust and vibrant as in Atlanta, but I've noticed in Philadelphia we tend to dwell on challenges and problems. Believe me, I saw the sausage making up close in Atlanta and it wasn't always perfect. But in Atlanta they tend to say, 'Bless your heart, let's not talk about that.' That's the only difference."
Claire Marrazzo Greenwood, a Greater Philadelphia Chamber of Commerce vice president who heads its CEO Council for Growth, shared some statistics that illuminated the scope and import of the biotech sector as a whole, as well as the cell and gene therapy part of it, where Philly is a particularly significant global player.
Philadelphia, she said, ranks second globally on the Journal of Life Sciences list of cities that are attracting entrepreneurs and skilled-technology graduates. In 2019 the Chamber launched an effort to support innovation in the region's cell- and gene-therapy hub. The nearly 60 companies now in the sector regionally are double the number four years ago. Philly is first among all American metro regions in National Institutes of Health funding for cell and gene therapy and the sector boasts $5 billion in new venture capital since 2019. (Here's a link to a recent Chamber study of the sector.)
The best-known success in the sector is Spark Therapeutics, whose CEO, Ron Philip, was the fourth panelist.
Just two days before the club event, Spark announced plans to build a half-billion-dollar gene therapy innovation center on the Drexel campus. Spark was the first company to get FDA approval to market a gene therapy. The therapy, called Luxturna, treats retinal disease. It stands as an early, shining example of how to take research emerging from Philadelphia's eds-meds nexus and turn that into a viable product.
"Luxturna was the first chapter of growth," Philip said. "Now, with this announcement, we are at work on the second chapter, which is expanding the platform to be able to do what we did with Luxterna for a variety of different diseases. We are focused on specific tissue types. Liver is one of our primary areas. We're also working on ophthalmology, beyond Luxterna. Finally, and it's early days, we're dabbling with work on Huntington’s disease and epilepsy."
Spark, which is up to 900 employees, was co-founded by Greenwood's brother, Jeff Marrazzo.
Philip explained the central idea of gene therapy, which is to identify the mutation in a person's genes that causes a disease, then find a way (there are a number of possible approaches) that attacks the mutation at, as he put it, a "foundational" level.
Wilson said the Science Center helps incubate and support firms in the biotech space, leveraging partnerships with the region's universities and hospitals, while also working to develop the workforce that the sector will need to thrive.
"I’ll give you an example," she said to the 70-plus people in attendance. "The other day I was walking from the parking lot to our office building at 36th and Market, and the new Drexel Health Sciences building rose up right behind us. And then right on the other side of Drexel is the new STEM high school. Literally you could see, 'Okay, there are students in that building. There are patients in this building behind me. There’s the next generation of talent in the building right behind that. Industry is right there, too.' We’ve got all of these programs and spaces to bring people together to figure out how to make that innovation process go faster. It is really inspiring to me."
Lao talked about how important it is to her that her company and the sector as a whole support the well-being of the city neighborhoods that surround University City:
"We are in West Philadelphia and we derive a tremendous amount of benefit from being there. There's the restaurants and the ease of transportation. And having eds and meds _ Wistar, Drexel, Penn, and CHOP - nearby for collaborations and conversations. But we also believe, particularly in West Philadelphia, that we must also create benefit and opportunity, being good citizens in that area. We try to be very thoughtful about where we order our supplies, where we buy our food, trying to support local people, not big chains."
Asked to name issues that keep them up at night, the panelists mentioned two big challenges:
1) When and how will the nation develop a reimbursement system for cell and gene therapies that will allow patients to afford the products to come?
2) How, as Philip said, to make the current optimism about the potential of cell and gene therapy doesn't leave patients with unrealistic expectations? The whole field is so new, he said, that the data simply don't exist yet to predict exactly for whom, how well or for how long any given therapy will work.