FDA’s CBER begins to regulate Gene Therapy products

In September 2000 FDA’s Center for Biologics Evaluation and Research (CBER) began to regulate the emerging market of human gene therapy products, which fall under the legal definition of a “biologic”.

Gene therapy research was 10 years old by this date.  The first clinical trial of a genetic therapy occurred on September 14, 1990, on a 4-year-old girl named Ashanthi DeSilva. She survived.  But then in September 1999, 18 year old Jesse Gelsinger died from a reaction to his experimental gene therapy treatment, which spurred and investigation that revealed not all trial adverse events were being reported. [1, 2]

It was reported that that since 1989 to September 2000, the FDA had “received about 300 requests from medical researchers and manufacturers to study gene therapy and to develop gene therapy products”, looking to find cures for genetic diseases. [3, 4]