Gene and engineered cell therapies hold great promise for treating rare genetic disorders, as well as chronic diseases. However, these therapies raise a number of challenges including regulatory barriers, equity in access to treatment, and affordability.
Somatic gene therapy alters or eliminates genes that cause disease in a patient without imparting these changes to future generations. This assessment will examine the challenges related to approval and use of these therapies, focusing on the latter stages of therapy development including drug approval, market introduction, and use.
National Research Council