Our Mission & History
The goals of the Gene Therapy Program at the University of Pennsylvania are to develop and commercialize transformative genetic-based therapeutics. These goals are driven by the unmet needs of patients with inherited genetic diseases.
The following Core Values, identified by our leadership, represent the Gene Therapy Program's principles and priorities.
Patient-focus
Engaging with the patient community and prioritizing their needs, values, and preferences
Trustworthiness
The quality of being reliable, responsible, and deserving of confidence
Fortitude
The strength to encounter adversity or difficulty with courage
Disruptive Innovation
An innovation that makes products and services more accessible and affordable, thereby making them available to a larger population. It is driven by enabling technology, an innovative business model, and a coherent value network.
Adaptability
The ability to adjust and flourish in a changing environment
Leaders in the development of innovative vector technology for nearly three decades
Neurologic
Eye Disease
Metabolic
Renal/Liver
Lung
Infectious Disease
Blood Disorders
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.