Updated US FDA Guidance For The Gene Therapy Industry: Key Changes And Areas Of Increased Focus

By Leyla Diaz, Ph.D., Senior Technical Specialist, Biosafety Testing Services, Rockville, MD

In January 2020, the United States Food and Drug Administration (FDA) released several new or updated guidance documents pertaining to human gene therapy products. The guidance documents address product development and clinical trial design in general, as well as for specific disease indications. Two guidance documents that address product development, safety and characterization are:

• Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up, which supersedes the guidance published in 2006
• Chemistry, Manufacturing and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs), which supersedes the guidance published in 2008

Several key changes that will impact developers of gene therapies are summarized below.