Pharmacologist U.S. Food and Drug Administration Silver Spring, Maryland
Development of gene therapy has unique challenges due to product complexity, unknown safety profiles, and cutting-edge testing methodologies. The presentation 1) overviews FDA current status of gene therapy, 2) discusses considerations and challenges from clinical pharmacology perspective with examples of CAR T cell therapy and AAV-based gene therapy, 3) provides FDA resources in gene therapy development.
Learn FDA current status of gene therapy.
Understand the differences between gene therapy products and conventional therapy products.
Learn about unique pharmacokinetic/pharmacodynamic characteristics of some gene therapy products (such as CAR-T cell therapy products and AAV-based gene therapy products).
Learn about considerations and challenges in clinical pharmacology assessment for gene therapy products.
Learn about FDA resourses for gene therapy products development.