FDA Releases Draft Guidances on Gene Therapies

The FDA unveiled six new draft guidances outlining its latest thinking on the rapidly evolving field of gene therapy, including two guidances that address the development of combination products and retinal disorder devices.

The draft guidances deal with chemistry, manufacturing and control information for investigational new drugs including combination products,  as well as retinal disorders, hemophilia, rare diseases, long-term follow-up, and testing of retroviral vector-based therapies.

The CMC guidance outlines the necessary components of a gene therapy IND, including labeling and environmental analysis, noting that sponsors must provide a list of all materials used in manufacturing and a description of the quality and control of the materials.

In a separate draft guidance, the agency deals with gene therapies for retinal disorders, which in some cases include an implanted device.