Value proposition

Catalyzing the development of transformative genetic-based therapeutics from concept to clinic.

Dedicated to discovery + translational research and led by Dr. James Wilson, the Gene Therapy Program (GTP) at the University of Pennsylvania 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's commitment to patients with rare diseases stems from his days as a graduate student and medical student at the University of Michigan between 1977 and 1984. He was discouraged by the lack of available treatments for rare disease patients, but optimistic that science could yield solutions with his initial focus being gene therapy. He completed his training in Internal Medicine at the Massachusetts General Hospital followed by a postdoctoral fellowship at the Whitehead Institute where he began his work in gene therapy.

After launching his faculty career in the Howard Hughes Medical Institute and the University of Michigan, he moved to the University of Pennsylvania in 1993. Jim’s laboratory has made seminal contributions to the technology of gene transfer and has paved the way for translation of these technologies into the clinic. He has published more than 550 papers and is named on more than 110 patents.

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

The Gene Therapy Program currently employs more than 350+ full-time employees with an annual operating budget based on funding from a diverse group of public and private sponsors.

Higher Education, AAV, Translational Research, Genome Editing, Capsid Engineering, Discovery Research, Vector Design, Biomedical Research, Rare Diseases, Clinical Research, GLP, Good Manufacturing Practice/GMP, Orphan Disease, Patient Registry, Gene Therapy, Good Laboratory Practice/GLP, Lipid Nanoparticles, Adeno-Associated Viruses, Genetic Medicine , and Genetic Therapies