The goals of the Gene Therapy Program at the University of Pennsylvania (GTP) are to develop and commercialize transformative genetic-based therapeutics. These goals are driven by the unmet needs of patients with inherited genetic diseases.
Led by Dr. James M. Wilson, GTP focuses on the development of next-generation gene transfer vectors and their application in the treatment of a variety of acquired and inherited diseases. Dr. Wilson began his work in gene therapy during his graduate studies at the University of Michigan more than 40 years ago and has been at the nexus of this emerging therapeutic area from its inception.
Dr. Wilson created the first and largest academic-based program in gene therapy after being recruited to the University of Pennsylvania in 1993. He initially focused on the clinical translation of existing gene transfer technologies but soon redirected his efforts to the development of second and third-generation gene transfer platforms. Since then, GTP has emerged as the go-to organization for public and private partners who want to participate in the gene therapy space.
The Wilson Lab discovered a family of viruses from primates called adeno-associated viruses (AAV) that could be engineered to be very effective gene transfer vehicles. These vectors have become the technology platform of choice and have set the stage for the recent resurgence of the field of gene therapy.
The following Core Values have been identified by our leadership and represent the Gene Therapy Program's principles and priorities.
Engaging with the patient community and prioritizing their needs, values, and preferences.
The quality of being reliable, responsible, and deserving of confidence.
The strength to encounter adversity or difficulty with courage.
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.
The ability to adjust and flourish in a changing environment.