Cellular and Gene Therapy

• The Center for Biologics Evaluation and Research (CBER) regulates human gene therapy products that introduce genetic material into the body to replace faulty or missing genetic materials. Cellular therapy products include cellular immunotherapies, and other types of both autologous and allogenic cells for certain therapeutic indications, including adult and embryonic stem cells. Human gene therapy refers to products that introduce genetic material into a person’s DNA to replace faulty or missing genetic material, thus treating a disease or abnormal medical condition.^[1]

FDA Resources

• Cellular & Gene Therapy Guidances
• Approved Cellular and Gene Therapy Products
• Therapeutic Cloning and Genome Modification

Updated by Kristin Stiner, December 2023