U.S. Department of Health and Human Services
National Institutes of Health
National Center for Advancing Translational Sciences
This page compiles third-party guides, resources and templates relevant for researchers and patient groups interested in learning more about gene therapy and developing a new gene therapy program.
Name
Source
Bespoke Gene Therapy Consortium (BGTC) Regulatory Playbook
NIH NCATS
FDA Accelerated Rare disease Cures (ARC) program
Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER)
FDA Rare Disease Innovation Hub
FDA Center for Biologics Evaluation and Research (CBER)
FDA CBER Cellular & Gene Therapy Guidances Website
FDA CBER
Gene Therapy Inside Out
FDA
Gene Therapy Patient Education Webinars
American Society for Gene and Cell Therapy (ASGCT)
Alliance for Regenerative Medicine Resources
Alliance for Regenerative Medicine (ARM)
A Guide to Gene Therapy
Global Genes