Other Resources

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