At PTC, we study human genes with a mission to treat rare genetic disorders. Our scientists are excited about the possibilities of gene therapy when it comes to treating rare diseases.

However, we understand that when people first think about gene therapy, they may have questions. What is it? How does it work? What problems can it solve?

That’s why, our team has developed this easy-to-understand video about how gene therapy works; and how PTC envisions gene therapy will help us treat rare genetic disorders, bringing more of life’s precious moments to people with rare diseases and their loved ones.