Gene therapy has evolved rapidly and may be poised to affect mainstream medicine. Gene therapy initially targeted incurable genetic diseases (e.g., metabolic diseases), but indications have now expanded, and now the most commonly used applications are to treat certain cancers. In response to the increase in approved gene therapy approaches, PCORI commissioned this landscape review. The purpose of this two-part report is to better understand the evidence supporting currently approved gene therapies and those that may be available in the near term in the United States.
Part I of the report focuses on adenovirus, adeno-associated virus vector-based (AAV) gene therapy, and clustered regularly interspaced short palindromic repeats (CRISPR). It addresses the description of the interventions, the context in which gene therapy is used, ongoing premarket and postmarket gene therapy studies, and current evidence for the use of gene therapy. View Part II of the report here.
The 2020 Nobel Prize in Chemistry was awarded to U.S.-based biochemist Jennifer Doudna and Germany-based microbiologist Emmanuelle Charpentier for their work in discovering and developing CRISPR, a genome editing tool. Find out more about the prize |
About the Emerging Technologies and Therapeutics Reports
The Emerging Technologies and Therapeutics Reports provide timely summaries of evidence supporting new drugs, devices, and other healthcare technologies that are recently in use or may be available in the near term in the United States. The documents also identify gaps that need to be addressed for the technologies or therapeutics to move forward.
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