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Watch: How Do Gene Therapies Work?

Gene therapy marks a new era in the battle against genetic diseases. Learn more in a short video.


Rather than treating symptoms, gene therapy can correct the underlying cause of disease. It represents a radical shift in how diseases – including rare diseases – are treated.

Learn more about cell and gene therapy, including a four-episode podcast series.

Read a news release about a hemophilia B gene therapy study shared at the European Association of Haemophilia and Allied Disorders (EAHAD) 2022 Annual Meeting.