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Pittsburgh’s next phase of growth in the life sciences and biotechnology didn’t come from a massive infusion of federal funding but from the ground up, whether it’s foundation funding or a startup with a big idea on how to treat a rare but devastating skin disease.
That was the case for Pitt BioForge, the University of Pittsburgh biomanufacturing center that’s under development along with a cell-therapy manufacturing plant that will be built there by Massachusetts-based ElevateBio. And it’s also the case for Krystal Biotech (Nasdaq: KRYS), whose founders were drawn to Pittsburgh after not being able to find suitable lab space in the San Francisco Bay Area.
Pittsburgh in general and the University of Pittsburgh and UPMC specifically are huge drivers of biomedical and biotech research, with Pitt being one of the largest recipients of medical research funding in the United States. But the region has often lost out to either coast when it comes to commercialization.
Leaders from BioForge, Krystal and the Richard King Mellon Foundation spoke Wednesday about the challenges and opportunities for the Pittsburgh region in biosciences as part of The Brookings Institution's nonpartisan national Election 2024 series. They were assembled at the Tepper School of Business at Carnegie Mellon University.
RK Mellon Foundation Director Sam Reiman said discussions around the challenges and opportunities led to the foundation giving $100 million in 2022 to create Pitt BioForge, a center that will develop ways to scale up and lower the cost of cell and gene therapy innovations. It was the largest grant for a single project in the foundation’s history.
“[It was] recognizing that the capital needed to get something like this off the ground was unlikely to come at that time from the private sector, and also unlikely to come from state or from federal sources,” Reiman recalled.
BioForge will support the goal to raise the region’s presence in biotechnology and especially the manufacturing process around cell and gene therapies that are developed at Pitt and UPMC.
Pitt BioForge’s leader, Ken Gabriel, said recent advances in cell and gene therapy are treating a range of diseases and conditions but come at a big price tag for individuals, between $500,000 to $5 million per therapeutic. What needs to happen are ways for the technology to scale as well as decline in production cost in a major way.
“What was interesting to me (about BioForge) was an opportunity to create that kind of driver to be an organization that is focused on creating the breakthroughs and innovation of precision biological medicines to speed their delivery, use and impact,” Gabriel said.
One Pittsburgh company that’s doing that now is Krystal Biotech, which went from a handful of scientists in 2016 to being publicly traded on the Nasdaq exchange and, more importantly, an FDA-approved treatment for a rare skin disease and potential therapies for other conditions and diseases in their pipeline.
Suma Krishnan, Krystal’s president, said a lack of lab space in the Bay Area led her to search the country.
“Pittsburgh, there was a lab that was readily available with some scientists with experience working with viral vectors so we decided to take over the lab and start the (needed) experiments and see,” she said.
It’s not easy. Biotech companies face incredible regulatory scrutiny, both in the United States and elsewhere in the world, as well as a need for large-scale funding. Krystal had not only a successful idea — born out by FDA approval — but also investors that believed in it because they knew the founders’ track record.
Krishnan said that it wouldn’t have been possible without all that but she praised the workforce Krystal found and attracted to Pittsburgh, from CMU, Penn State and elsewhere. She said FDA’s inspection team was impressed with the quality of the workforce and, with the plans to take the product into Europe, with a Dutch inspection team as well. There are plans to make its gene therapy available in Japan, too.
“It’s a global disease, so we want to make sure that a product that’s made in Pittsburgh can be distributed to an approved in the rest of the world,” Krishnan said.