Traditional approaches to treating patients using medicines and surgery have improved the lives of people throughout the world but there are many diseases, perhaps even most of them, for which these traditional approaches modulate the course of the disease but do not provide a cure. This shortcoming is particularly apparent for treating monogenic diseases, diseases caused by mutations in single genes that a person is born with. It is estimated that there are greater than 6,000 such diseases affecting over 350 million people worldwide.
Cells and genetically engineered cells have fundamentally different properties than medicines and surgery—they are “living drugs” that can heal and replace damaged tissues or diseased organs. These properties mean that they have the potential to provide curative therapies to a range of diseases that currently have no cure.