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Targeted gene-editing therapies offer a huge potential for treating genetic diseases.

Did you know that there are two ways to deliver CRISPR/Cas9 gene-editing tools to the correct cells in the body? They are known as ex vivo and in vivo, and both methods are intended to precisely change a person’s DNA to target the specific cause underlying a disease. Learn more about these two methods and how ex vivo gene-editing therapies are prepared and manufactured.

At Vertex Pharmaceuticals, we believe true scientific transformation happens at the intersection of human biology and medical innovation. Our scientists are researching ex vivo gene-editing therapies, where the process starts with the collection of cells in a person’s body and ends with returning the gene-edited cells back to the same person so they can find their home and get to work inside the body. The goal? Impacting serious diseases for which no curative therapies exist.

About this video series

This series shares the science behind some of the tools and technologies we use in our research and clinical development. As our scientists continue to make progress, we want to spread awareness about the innovative technology used behind the scenes in our labs. Check out some of the other tools in our toolbox that we are using to advance toward our goals: