Cellular & Gene Therapy Guidances (Food and Drug Administration)

 

• Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial; Draft Guidance for Industry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

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

• Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products; Draft Guidance for Industry
  3/2022

• Human Gene Therapy Products Incorporating Human Genome Editing; Draft Guidance for Industry
  3/2022

• 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