The original goal of gene therapy was to deliver a single administration that would maintain therapeutic effectiveness over a patient’s lifetime. This ‘one and done’ model appears to have worked for some gene therapies, with promising follow-up data evidenced. However, in some instances, including gene therapies for hemophilia, a single dose may not be enough, and redosing may be required.

In this article, our gene therapy experts at Health Advances, Parexel’s commercial strategy group, outline considerations for ’one-and-done’ versus redosing therapies.