FDA issues new gene therapy guidances

July 12, 2018

FDA’s Center for Biologics Evaluation and Research (CBER) issued a suite of six scientific guidance documents intended to serve as a framework for gene therapies.

The agency hopes the guidances will “serve as the building blocks of a modern, comprehensive framework for how we’ll help advance the field of gene therapy while making sure new products meet the FDA’s gold standard for safety and effectiveness.”

The documents are being issued in draft form so that the FDA can solicit public input on the new policies. The first three guidance documents focus on gene therapy products for hemophilia, rare diseases and retinal disorder. The next focus on CMC info for new drug applications, follow-up observational studies collecting data on adverse events and the testing of retroviral vector-based therapies.

Read the guidances here:

draft guidance on gene therapy products that are targeted to the treatment of hemophilia

Human Gene Therapy for Retinal Disorders guidance

Human Gene Therapy for Rare Diseases guidance

Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs)

Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus (RCR) during Product Manufacture and Patient Follow-up
Long Term Follow-Up After Administration of Human Gene Therapy Products