University of Pennsylvania gene therapy pioneer James M. Wilson has been using adeno-associated viral (AAV) vectors in his work for decades. In traditional gene therapy, they act as transporters, carrying the normal version of a gene to a desired cell.
“For someone with genetic blindness, we could deliver a gene to the retina. For someone with muscular dystrophy, we could deliver a gene to the muscle,” says Wilson, director of the Gene Therapy Program and the Orphan Disease Center at the Perelman School of Medicine. “A virus is the most efficient way to do this.”
Viruses typically shuttle their own genes into the cells and cause a person to get sick, sticking around for as long as those cells do. But what if, rather than leading to illness, these vectors instead brought antibodies that could counter SARS-CoV-2?
In collaboration with pharmaceutical company Regeneron, Wilson is working on this approach, which has potential to stop the coronavirus from spreading, as an alternative or supplement to the current vaccines being rolled out. In November, the FDA issued an Emergency Use Authorization (EUA) for a cocktail of two of Regeneron’s lab-made antibodies, casirivimab and imdevimab, to treat COVID-19, with IV infusion as the stipulated treatment method. Though that EUA does not apply to Wilson’s approach, he says he thinks it should make getting future approval for the AAV technique easier.
Penn Today spoke with Wilson about the implications of this research for the current pandemic.
James M. Wilson is director of the Gene Therapy Program, the Rose H. Weiss Professor and director of the Orphan Disease Center, and a professor of medicine and pediatrics at the Perelman School of Medicine at the University of Pennsylvania.