Deliver the Product

At CHOP, we understand the challenges of commercialization, particularly for orphan (rare) diseases. Our internal expertise is critical to our external partnerships. We have the data and in-depth knowledge necessary to fuel future breakthroughs, and we explore all funding avenues, including partnerships with pharma and investments in spinoff companies.

Our deliberate, strategic investments in innovation have paid off big, and we’ve learned a lot along the way. Our work has helped establish Philadelphia as a hub for cell and gene therapy, and we’ve powered some of the most significant advances in this exciting field.

Partnering with Pharma

First-ever Cell Therapy to Receive FDA Approval

In August 2017, the FDA approved the use of chimeric antigen receptor T-cell (CAR-T) therapy for the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) in children and young adults. Pioneered through CHOP’s collaboration with the University of Pennsylvania and licensed by Novartis, Kymriah is an engineered cell therapy in which a patient’s own reprogrammed T cells locate and kill cancerous cells. The FDA’s landmark approval of this CAR-T cell gene immunotherapy is the result of five years of multisite, global clinical trials supervised by lead investigator Stephan Grupp, MD, PhD, Director of the Cancer Immunotherapy Program at CHOP. The approval of Kymriah has shifted the paradigm for pediatric cancer treatment — and at CHOP, more cell therapies are on the horizon.

Learn What’s Next for CAR-T Cell Therapy

Investing in Innovation

Spark Therapeutics Commercializes Gene Therapy for Genetic Blindness

Almost 20 years ago, Katherine High, MD, then Director of the Center for Cellular and Molecular Therapeutics at CHOP, aspired to using gene therapy in the treatment of inherited retinal dystrophies, a group of eye disorders characterized by retinal degeneration due to two mutations in the RPE65 gene. After a decade of research, High proved the potential of gene therapy in the treatment of retinal dystrophies. In an effort to propel her research and garner the funding and talent necessary to quickly commercialize and deliver this life-changing therapy, CHOP helped found Spark Therapeutics, a Philadelphia biotechnology company. In less than one year, Spark tested its blindness therapy in clinical trials that ultimately illustrated the treatment’s game-changing potential. In 2017, Spark produced Luxturna, the first U.S. Food and Drug Administration-approved gene therapy for an inherited form of blindness. Delivered by subretinal injection, Luxturna provides retinal cells a working copy of the mutated RPE65 gene. This prompts the cells to make the RPE65 protein, which allows the visual cycle to continue and light to be converted to electrical signals that can be interpreted by the brain.

Learn More About Luxturna and Spark Therapeutics

Licensing a New Product

CHOP Team Partners with Medical Equipment Manufacturer

Unlike other protective covers for children with intravenous (IV) lines, the See-IV has a clear plastic window through which a nurse can see the IV area. Designed by Michele Davey, RN, and CHOP collaborators, the See-IV makes frequent IV checks more efficient for staff and less disruptive for patients. With the guidance and support of CHOP’s Office of Entrepreneurship and Innovation (OEI), the team developed and licensed See-IV through Medline, a medical equipment manufacturer.

Learn More About See-IV and the Office of Entrepreneurship and Innovation

Spinning Off a Startup Company

Simulated Driving Software Builds on Decades of Research

Diagnostic Driving’s simulated driving software rapidly identifies dangerous driving behaviors, categorizes drivers by crash risk and delivers personalized online coaching. Developed by Flaura Winston, MD, PhD, Founder and Scientific Director of the Center for Injury Research and Prevention (CIRP) at CHOP, and Venk Kandadia, MPH, former CIRP clinical research project manager, Diagnostic Driving’s scientifically-grounded software results from decades of CIRP research on driver safety. With the support of OEI and a seed investment from CHOP, Diagnostic Driving was spun out as a standalone company in 2015.

Learn More About Diagnostic Driving