Bringing medical innovations to life

Developing new treatments requires years of research and thousands of hours of dedicated effort. Even so, some treatments never meet the strict safety and efficacy standards for approval by the U.S. Food and Drug Administration (FDA).

Penn Medicine is not only one of the world’s top institutions for biomedical research. Our culture and collaborative organization are committed to transforming breakthroughs in the lab into new, lifesaving treatments. We’ve often been the first to do just that. Our researchers pioneered CAR T cell therapy, the groundbreaking cancer treatment developed here and first approved by the FDA in 2017. CAR T cell therapy has since been used to treat thousands of patients around the world, helping many reach remission. We also achieved the first FDA approval for a gene therapy to treat blindness and played a critical role in developing the mRNA vaccine technology that revolutionized the fight against COVID-19. These are just a few examples of how our research is creating tomorrow’s cures today.

Penn Medicine’s Office of Clinical Research and the Penn Center for Innovation play critical roles in the journey of bringing new medicines to patients, offering faculty unparalleled regulatory and commercial expertise to move ideas forward. By fostering partnerships with biotech companies and equipping researchers with the tools they need to navigate regulatory standards, we bridge the gap between discovery and real-world impact.

Penn Medicine is a global leader in basic, translational and clinical research and an epicenter for the development of new treatments and technologies that deliver new medicines to patients.

Since 2013, our researchers have made substantial, essential contributions— whether through foundational research or clinical trials—to at least 41 FDA approvals, and still counting.

Almost half of these approvals were first-in-class for the type of disease they treat. Even more noteworthy, Penn has led discoveries of the first approved treatments of entire new categories of treatments, including the first-ever approved personalized cellular therapy (CAR T cell therapy), the first-ever approved mRNA vaccines, and the first-ever approved gene therapy. We continue to drive basic science discoveries and are committed to translating the latest innovations in genetics, immunology, and cell and gene therapy into additional approved medicines.

Here are some of our recent approved treatments:

• Tisagenlecleucel: The first personalized gene therapy approved in the United States
• Iobenguane i 131: The first and only treatment option for adults and adolescents with malignant, recurrent, or unresectable pheochromocytomas and paragangliomas
• Gilteritinib: The first tyrosine kinase-inhibitor (TKI) drug for the treatment of patients with relapsed/refractory acute myeloid leukemia (AML) and mutation of FMS-like tyrosine kinase 3 (FLT3)
• Selinexor: The first oral, selective exportin 1 (XPO1) inhibitor for combined use with dexamethasone to treat relapsed/refractory multiple myeloma (RRMM)
• Belantamab mafodotin-blmf: The first antibody–drug conjugate that selectively targets and kills myeloma cells
• Belzutifan: The first oral hypoxia-inducible factor (HIF)-2α inhibitor for treating tumors associated with von Hippel-Lindau disease
• Pafolacianine: The first receptor-targeted, intraoperative, fluorescence-imaging agent capable of illuminating ovarian cancer and detection of lung cancer lesions
• Teclistamab-cqyv: The first bispecific T cell engager antibody for the treatment of patients with relapsed or refractory multiple myeloma

Penn Medicine is making significant advancements in cancer treatment, promoting innovative approaches to cancer therapy worldwide. Our efforts have led to the development of numerous new immunotherapies and innovative therapies.

The increase in treatment options is driven by a deeper understanding of how cancer develops and grows in the body, and especially the immune system’s role in combating cancer. These diverse approaches include medicines that weaken an already-weakened cancer cell, such as PARP inhibitors like olaparib, which make certain breast, ovarian, and prostate cancers that have BRCA mutations, unable to survive. Cancer treatments clinically proven at Penn can also make it easier to remove tumors surgically, as with pafolacianine, a fluorescent dye that helps doctors see and precisely remove tumors during surgery. With each new treatment approval, Penn Medicine’s integrated approach—from the laboratory to the clinic—continues to speed the delivery of effective therapies to cancer patients.

Learn more about how Penn Medicine research is powering new treatments.