Cell and gene therapies have the potential to change the way rare diseases are treated, offering novel and more effective treatments for patients with limited options. This article explores the various options available from the government to help rare disease drug...
Figure 1. Overview of the biodistribution study of cell/gene therapy (CGT) product. The CGT product is injected into mice; tissue samples are harvested and DNA is isolated from all samples. DNA is then quantified and used as a template in quantitative polymerase chain...