Our Mission & History

The goals of the Gene Therapy Program, to develop and commercialize transformative genetic-based therapeutics, are driven by the unmet needs of patients with inherited genetic diseases.

Leaders in the development of innovative vector technology for nearly three decades

neuro

Neurologic

eye

Eye Disease

metabolic

Metabolic

liver

Renal/Liver

lungs

Lung

infection

Infectious Disease

blood

Blood Disorders

neuromuscular

Neuromuscular

Affiliations

Orphan Disease Center

The Orphan Disease Center was formed in 2011 with a generous gift from philanthropist and Wharton graduate, George A. Weiss. The Center, which is the first of its kind, was created to facilitate translational research and therapy development to address the prevalence of certain orphan diseases that affect a marginalized population of individuals internationally.

Penn Center for Innovation

The Penn Center for Innovation (PCI) is a premier university innovation, venture creation, and commercialization center. PCI actively fosters innovation within the Penn community and provides essential support to protect and commercialize Penn’s intellectual property portfolio. PCI is also active in developing external commercial partnerships worldwide.