Gene therapy is a new kind of treatment for a new era of medicine. The goal of gene therapy is to add a new copy of a missing or malfunctioning gene. Gene therapies aim to target the underlying cause of genetic disease, and they offer the opportunity to treat a wide range of severe inherited diseases that previously had few treatment options.
How much gene therapy is needed – the amount of the gene therapy dose – depends on the nature of the disease being treated, and how many and what type of cells are affected. In a systemic disease, meaning it affects the entire body, the therapy needs to target and repair many cells across the entire body.
This isn’t a 200-milligram pill. This is sometimes trillions of corrected genes that need to be delivered precisely where they are needed, and with sufficient quantity to generate a clinically relevant response over time.
From the science to the manufacturing, to the dosing, gene therapies are different from what we’re used to thinking about medicines. Go inside the science with this video to learn more about gene therapy and gene therapy dosing.
