Gene therapy products now have the potential to cure severe conditions such as cancer and fundamentally alter the trajectory of many other severely disabling diseases by engineering the correct functionality of cells or genes.
In the article, Paula and Björn share their insights on the selection of viral vectors and the pros and cons related to the different types of vectors. When developing gene therapies, one major hurdle is the lack of in vivo models to generate safety data. Paula and Björn also explore the decisions that needs to be considered when moving a potential gene therapy from the pre-clinical phase to first in human studies. The article also covers the clinical evidence generation and what impact the usually small patient populations have on the safety data.