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Treatment options expand in robust gene therapy pipeline



The latest gene therapy pipeline update shows a growing number of innovative treatments in development for a range of conditions, including sickle cell disease, severe hemophilia A, hemophilia B, and different cancers such as acute lymphoblastic leukemia.


Our periodic gene therapy pipeline update report shares information about treatments that may come up for review and approval by the U.S. Food and Drug Administration (FDA) in the near term.


Treatments in the drug pipeline change based on the success – or lack thereof – of clinical trials and other important data. We will continue to share updates to the gene therapy pipeline periodically.