Resources

This page is a compilation of third-party guides, resources and templates that are highly relevant to researchers and patient groups that want to learn more about gene therapy and how to develop a new gene therapy program.

Name Source
FDA Gene Therapy Guidances
FDA CBER Cellular & Gene Therapy Guidances Website Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER)
Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) FDA CBER
Preclinical Assessment of Investigational Cellular and Gene Therapy Products FDA CBER
Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products FDA CBER
Patient-Friendly Resources on Gene Therapy
Gene Therapy: Your Questions Answered National Organization for Rare Disorders (NORD)
A Guide to Gene Therapy Global Genes
Gene Therapy Inside Out FDA
Gene Therapy Educational Resources Genetic Science Learning Center
Gene Therapy Patient Education Webinars American Society for Gene and Cell Therapy (ASGCT) and NORD
Genoterapia (en espaƱol) Mayo Clinic
Standards and Best Practices for New Programs
Regenerative Medicine Standards Landscape Standards Coordinating Body
Perspectives on Best Practices for Gene Therapy Programs Human Gene Therapy
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