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The first gene therapy treatment, Kymriah, was approved only three years ago. Since then, with greater advances in scientific methods and emergence of gene editing techniques such as CRISPR (clustered regularly interspaced short palindromic repeats), the number of treatments, as well as the conditions that could be treated with gene therapies, have continued to grow.
In our periodic gene therapy pipeline update report, we share information about treatments that may come up for review and approval by the U.S. Food and Drug Administration (FDA) in the near-term.
Such treatments could offer a cure for previously untreatable conditions. They could also help change the way care is delivered for chronic, inherited conditions like hemophilia and sickle cell disease, which in the past required lifelong treatments.
In 2020 alone there are several possible treatments awaiting FDA approval to treat different types of hemophilia, and cancers.
Treatments in the pipeline change often 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.
This document contains references to brand-name prescription drugs that are trademarks or registered trademarks of pharmaceutical manufacturers not affiliated with CVS Health.