Project Summary

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.

Report Part I (Published March 2019)

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.

Report Part II (Published June 2019)

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.

Read Report (Part I) Read Report (Part II)

Issue Brief (Published July 2019)

This issue brief discusses approved therapies and those on the horizon, as well as the challenges and opportunities ahead.

View Issue Brief

B-cell Lymphoma and Non-Hodgkin’s Lymphoma Treatment: How Do Participants of Chimeric Antigen Receptor T-cell Therapy Research Studies Compare With Patients in the US Population? (Published June 2020)

Human gene therapy has evolved rapidly, and chimeric antigen receptor T-cell (CAR-T) therapy appears to be a promising treatment for B-cell lymphoma and non-Hodgkin’s lymphoma. This report compares participants of CAR-T therapy research studies to individuals living with the diseases in the US population.

Read Report

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.

Journal Citations

Project Information

RAND Corporation

Key Dates

July 2019


Project Status
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Last updated: March 4, 2022