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 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. View Part I of the report here.
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.
The Patient-Centered Outcomes Research Institute sends weekly emails about opportunities to apply for funding, newly funded research studies and engagement projects, results of our funded research, webinars, and other new information posted on our site.
