Academia and industry form powerful partnerships for impact
Since 2018, the Penn Medicine Co-Investment program has spurred faculty innovation, created jobs, and scaled up ideas into large-scale clinical studies.
How did Philadelphia earn the nickname “Cellicon Valley”?
Along with a lot of groundbreaking research and world’s-first discoveries in cell and gene therapies, the Penn Center for Innovation has played a key role in facilitating the development of faculty ideas and discoveries into new products and businesses, including commercially available therapies. The center helps researchers turn their ideas and discoveries into opportunities that can be licensed to existing businesses for commercial development, or to launch startup companies spun out from Penn.
In certain cases, the Penn Medicine Co-Investment Program has provided crucial financial support in bringing these new companies to life. Since the program was established in 2018 through an investment from the health system, it has supported 14 spinout companies to commercialize technologies emerging from Penn Medicine labs.
Resources like the Penn Medicine Co-Investment Program and the Penn Center for Innovation are key connectors in a virtuous cycle of research discovery, helping to bring ideas from academic labs that often rely on federal and philanthropic funding, into a phase of larger-scale clinical testing and commercial development.
“NIH funding, academic research, philanthropic funding, and corporate funding working together really does change lives,” said Carter Caldwell, the co-investment program’s director.
An investment in the Philadelphia region
Academic institutions sometimes invest in companies to generate returns, but that wasn’t Penn’s goal. “Penn said, ‘We want to use our money to spur faculty innovation, attract capital to Philadelphia, create jobs, and build up Philadelphia as a nexus for cell and gene therapy,’” Caldwell explained.
The return on investment has been significant for the region: The Penn spinouts have created nearly 500 jobs in Philadelphia and attracted $1.8 billion of private investment.
More than 100 investors have supported Penn’s spinouts, and high-profile venture capital firms have approached the University unsolicited, drawn by its reputation for innovation.
“There is very important funding that plants the seed in faculty members’ labs,” Caldwell said. “And that is ultimately what brings research to the finish line of possibly becoming a new treatment for patients. But then it requires significant additional money to perform the rigorous clinical trials necessary to get over the finish line of FDA approval, and that’s where private capital comes into play.”
Startup companies seeing success and scale of impact
The success stories continue to grow.
Tmunity Therapeutics, which was launched by Penn researchers in 2015, was one of the first startups to receive Penn Medicine Co-Investment funds. It was later acquired by Kite, a Gilead Company. Kite recently funded a successful dual-target glioblastoma trial and will also fund the next phase of an “armored” CAR T trial at Penn—advancing two CAR T approaches that have already led to exciting results for hard-to-treat cancer patients.
Interius BioTherapeutics, a spinout from the lab of Saar Gill, MD, PhD, an associate professor of Hematology-Oncology, is seeing promising early results with its in vivo CAR gene therapy for B-cell malignancies. Meanwhile, Cabaletta Bio, which sprung from the labs of Michael Milone, MD, PhD, and former Penn faculty member Aimee Payne, MD, PhD, is applying CAR T cell therapy to autoimmune diseases.
Then there’s Capstan Therapeutics, whose scientific co-founders include a multidisciplinary lineup of eight Penn faculty working on an in vivo CAR T platform that harnesses the power of messenger RNA (mRNA). The method adapts the technology used to deliver mRNA into cells that formed the basis of successful vaccines against COVID-19—and led to a 2023 Nobel Prize for Drew Weissman, MD, PhD, and Katalin Karikó, PhD, who spent years discovering and rigorously testing their modified mRNA technology, years before the global pandemic. It uses mRNA specifically to deliver instructions to create CAR T cells in vivo that target the cells behind a disease. The in vivo approach, modifying cells inside the body instead of extracting, engineering, and reinfusing them, could potentially revolutionize the power of CAR T cells—saving time, money, and making these personalized therapies more accessible.
Capstan was founded based on initial lab results published in 2022 that used mRNA to create CAR T cells that targeted fibrosis involved in heart disease, by a team led by Jonathan Epstein, MD, now dean of the Perelman School of Medicine and executive vice president of the University for the Health System. That early work was funded by a combination NIH grants and a private foundation and philanthropy.
A raft of news showing the promise of the approach came this June: A Capstan-sponsored Phase 1 clinical trial began testing this approach for autoimmune disease, in healthy volunteers. Some of the team members published a high-profile study in Science on findings that the platform could successfully target B cells in autoimmune disease in preclinical animal studies.
Then, pharmaceutical company AbbVie announced it will acquire Capstan for $2.1 billion to further develop the Penn spinout’s technology.
“The economic and lifesaving impact of basic laboratory research is so important—our experience with this technology that literally engineers immune cells in the body to create a treatment is just one example that shows how basic science is often the catalyst for so much more,” Epstein said. “We depend on federal funding, philanthropy, and other support to get promising scientific ideas off the ground. And as discovery science moves ahead, these investments spur significant economic growth while they extend and save lives.”