Other 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 español)	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

Rare Pediatric Disease (RPD) Designation Request for AAV9-hPCCA

This pdf file contains the RPD designation request for AAV9-hPCCA (NCATSBL-0746) and associated communications between NCATS and FDA OOPD.

March 21, 2023

Orphan Drug Designation (ODD) Request for AAV9-hPCCA

This pdf file contains the ODD request for AAV9-hPCCA (NCATSBL-0746) and associated communications between NCATS and FDA OOPD.

March 21, 2023

Rare Pediatric Disease (RPD) Designation Request Template Read More

February 27, 2023

Orphan Drug Designation (ODD) Request Template Read More

February 27, 2023

National Institutes of Health