My name is Larissa Lapteva, and I am the Associate Director
in the Division of Clinical Evaluation, Pharmacology and Toxicology in
the Office of Tissues and Advanced Therapies in the Center for Biologics Evaluation and Research
at the Food and Drug Administration.
Today, I will give you an overview of the regulatory approach to the development of
cellular and gene therapies aiming to treat diseases, in serious medical conditions.
In the United States, cellular and gene therapy products are regulated
by the Food and Drug Administration (or FDA).
At the FDA, the oversight of medical products for human and veterinary use is accomplished
through the work of various centers.
For example, there is a Center for Biologics, a Center for Drugs, and a Center for Devices,
The expertise for the development and regulation of cellular and gene therapies
aimed to treat diseases currently resides in the Center for Biologics Evaluation and Research,
specifically in the Office of Tissues and Advanced Therapies.
When a new product is developed, a multidisciplinary team from the FDA side
supports that product development program.
The team would often include pharmacologists, toxicologists, chemists, and (for example)
for gene therapies it may include biologists with backgrounds in genetics, as well as physicians,
statisticians, and other professionals whose input is useful
for successful development of a new treatment.