The Association of Clinical Research Professionals

Understanding Regulatory Guidance for Gene Therapy Development

It has been a big year for gene therapy. In the past 12 months, three gene therapy products received approval from FDA. In July 2018, FDA Commissioner Scott Gottlieb released a statement on the FDA’s efforts and commitment to advance gene therapy. The statement included the announcement of six new or revised guidance documents related to the development of gene therapy products. In this presentation, Dr. Vaughn will review Commissioner Gottlieb’s statement, provide an overview of the six guidance documents, and discuss the implications for clinical development of new gene therapy products.


This program is a recorded session from ACRP 2019