FDA Details Policies on Gene Therapies in Seven Guidances

The FDA unveiled seven guidances that clarify the agency’s policies for sponsors of gene therapy products for specific disease areas.

Commissioner Stephen Hahn said the FDA aims to encourage innovation in the rapidly growing gene therapy market. The agency has so far approved just four gene therapies but it currently has  900 gene therapy INDs on file.

The new guidance package includes separate guidances on gene therapies for rare diseases, hemophilia and retinal disorders, with considerations on chemistry, manufacturing and controls, preclinical studies and clinical trials for each therapeutic area.