Cellular and Gene Therapy Guidances

Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products; Draft Guidance for Industry
11/2024

Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products; Draft Guidance for Industry
4/2024

Considerations for the Use of Human-and Animal-Derived Materials in the Manufacture of Cell and Gene Therapy and Tissue-Engineered Medical Products; Draft Guidance for Industry
4/2024

Human Gene Therapy Products Incorporating Human Genome Editing; Guidance for Industry
1/2024

Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products; Guidance for Industry
1/2024

Potency Assurance for Cellular and Gene Therapy Products; Draft Guidance for Industry
12/2023
For a high-level overview of this guidance document, please view this recorded webinar featuring Dr. Matthew Klinker, Cell Therapy Branch 2 Chief, Office of Cellular Therapy and Human Tissues, Office of Therapeutic Products, CBER. 

Manufacturing Changes and Comparability for Human Cellular and Gene Therapy Products; Draft Guidance for Industry
7/2023
For a high-level overview of this guidance document, please view this recorded webinar featuring Dr. Andrew Byrnes, Gene Transfer & Immunogenicity Branch Chief, Office of Gene Therapy CMC, Office of Therapeutic Products, CBER.

Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial; Guidance for Industry
11/2022

Human Gene Therapy for Neurodegenerative Diseases; Guidance for Industry
10/2022

Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations; Guidance for Industry
9/2021

Manufacturing Considerations for Licensed and Investigational Cellular and Gene Therapy Products During COVID-19 Public Health Emergency; Guidance for Industry
1/2021

Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs); Guidance for Industry
1/2020

Long Term Follow-up After Administration of Human Gene Therapy Products; Guidance for Industry
1/2020

Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up; Guidance for Industry
1/2020

Human Gene Therapy for Hemophilia; Guidance for Industry
1/2020

Human Gene Therapy for Rare Diseases; Guidance for Industry
1/2020

Human Gene Therapy for Retinal Disorders; Guidance for Industry
1/2020

Evaluation of Devices Used with Regenerative Medicine Advanced Therapies; Guidance for Industry
2/2019

Expedited Programs for Regenerative Medicine Therapies for Serious Conditions; Guidance for Industry
2/2019

Regulatory Considerations for Human Cells, Tissues, and Cellular and Tissue-Based Products: Minimal Manipulation and Homologous Use; Guidance for Industry and Food and Drug Administration Staff
Updated: 12/2017

Same Surgical Procedure Exception under 21 CFR 1271.15(b): Questions and Answers Regarding the Scope of the Exception; Guidance for Industry
11/2017

Deviation Reporting for Human Cells, Tissues, and Cellular and Tissue-Based Products Regulated Solely Under Section 361 of the Public Health Service Act and 21 CFR Part 1271; Guidance for Industry
9/2017

Recommendations for Microbial Vectors Used for Gene Therapy; Guidance for Industry
9/2016

Design and Analysis of Shedding Studies for Virus or Bacteria-Based Gene Therapy and Oncolytic Products; Guidance for Industry
8/2015

Considerations for the Design of Early-Phase Clinical Trials of Cellular and Gene Therapy Products; Guidance for Industry
6/2015

Determining the Need for and Content of Environmental Assessments for Gene Therapies, Vectored Vaccines, and Related Recombinant Viral or Microbial Products; Guidance for Industry
3/2015

Guidance for Industry: BLA for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System
3/2014. (This guidance finalizes the draft guidance of the same title dated June 2013.)

IND Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System – Guidance for Industry and FDA Staff
3/2014. (This guidance finalizes the draft guidance of the same title dated June 2013.)

Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products
(This guidance finalizes the draft guidance entitled “Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products” dated November 2012) 11/2013

Guidance for Industry: Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage
12/2011. (This guidance finalizes the draft guidance of the same title dated July 2007.)

Guidance for Industry: Clinical Considerations for Therapeutic Cancer Vaccines
10/2011. (This guidance finalizes the draft guidance of the same title dated September 2009.)

Guidance for Industry: Potency Tests for Cellular and Gene Therapy Products
1/2011. (This guidance finalizes the draft document of the same name, dated October 2008.)

Guidance for Industry: Cellular Therapy for Cardiac Disease
(This guidance finalizes the draft guidance entitled “Guidance for Industry: Somatic Cell Therapy for Cardiac Disease” dated March 2009 (April 2, 2009, 74 FR 14992). 10/2010.

Guidance for Industry: Considerations for Allogeneic Pancreatic Islet Cell Products
9/2009

Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Somatic Cell Therapy Investigational New Drug Applications (INDs)
4/2008

Eligibility Determination for Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products; Guidance for Industry
8/2007

Guidance for Industry: Guidance for Human Somatic Cell Therapy and Gene Therapy
3/1998