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 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. Because the literature base across all of the interventions is so large, we split the content into 2 parts.
Part I: Adenovirus, Adeno-Associated Virus, and Clustered Regularly Interspaced Short Palindromic Repeats
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.
Part II: Chimeric Antigen Receptor-T cell (CAR-T), Autologous Cell, Antisense, RNA Interference (RNAi), Zinc Finger Nuclease (ZFN), Genetically Modified Oncolytic Herpes Virus
Part II of the report focuses on chimeric antigen receptor T cell (CAR-T); autologous cell; zinc finger nuclease (ZFN); antisense; ribonucleic acid interference (RNAi); and oncolytic viral therapy replication-competent, attenuated derivative of herpes simplex virus type 1 therapies.
This four-page document spotlights information from the Landscape Review and Evidence Map of Gene Therapy. The issue brief discusses approved therapies and those on the horizon, as well as the challenges and opportunities ahead.
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.