Cellular & Gene Therapy Guidance Documents

• Draft Guidance for Industry and FDA Staff: Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System (PDF - 133KB)
  6/2013. (This guidance document is for comment purposes only.)
• Draft Guidance for Industry: Biologics License Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System (PDF - 328KB)
  6/2013. (This guidance document is for comment purposes only.)
• Draft Guidance of Industry: Preclinical Assessment of Investigational Cellular and Gene Therapy Products (PDF - 168KB)
  11/2012. (This guidance, when finalized, will supersede the recommendations in section VIII in the final guidance entitled “Guidance for Industry: Guidance for Human Somatic Cell Therapy and Gene Therapy” dated March 1998 (Ref. 1).
• Guidance for Industry: Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee Cartilage (PDF - 157KB)
  12/2011. (This guidance finalizes the draft guidance of the same title dated July 2007.)
• Guidance for Industry: Clinical Considerations for Therapeutic Cancer Vaccines (PDF - 75KB)
  10/2011. (This guidance finalizes the draft guidance of the same title dated September 2009.)
• Guidance for Industry and FDA Staff: Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications. (PDF - 104KB)
  6/2011
• Guidance for Industry: Potency Tests for Cellular and Gene Therapy Products (PDF - 311KB)
  1/2011. (This guidance finalizes the draft document of the same name, dated October 2008.)
• Guidance for Industry: Cellular Therapy for Cardiac Disease
  10/2010. (This guidance finalizes the draft guidance entitled “Guidance for Industry: Somatic Cell Therapy for Cardiac Disease” dated March 2009 (April 2, 2009, 74 FR 14992).
• Draft Guidance for Industry: Assay Development for Immunogenicity Testing of Therapeutic Proteins (PDF - 161KB)
  12/2009
• Guidance for Industry - Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic Reconstitution for Specified Indications (PDF - 462KB)
  10/2009
• 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 Gene Therapy Investigational New Drug Applications (INDs)
  4/2008
• 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
• Draft Guidance for Industry: Validation of Growth-Based Rapid Microbiological Methods for Sterility Testing of Cellular and Gene Therapy Products
  2/2008
• Guidance for Industry: Eligibility Determination for Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products
  8/2007
• Guidance for Industry: Gene Therapy Clinical Trials - Observing Subjects for Delayed Adverse Events
  11/2006
• Guidance for Industry: Supplemental Guidance on Testing for Replication Competent Retrovirus in Retroviral Vector Based Gene Therapy Products and During Follow-up of Patients in Clinical Trials Using Retroviral Vectors
  11/2006
• Guidance for Industry: Guidance for Human Somatic Cell Therapy and Gene Therapy
  3/1998
• Guidance for the Submission of Chemistry, Manufacturing, and Controls Information and Establishment Description for Autologous Somatic Cell Therapy Products (TXT - 59KB)
  1/1997