FDA working to advance development of gene therapies

FDA Commissioner Scott Gottlieb, MD, announced Wednesday that the agency is releasing six guidance documents that are "intended to 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." Three of

FDA Commissioner Scott Gottlieb, MD, announced Wednesday that the agency is releasing six guidance documents that are "intended to 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." Three of the new gene therapy draft guidance documents focus on specific disease categories: hemophilia, retinal disorders, and rare diseases. In addition, FDA is providing updates to three existing guidances that address manufacturing issues related to gene therapy. These include "Chemistry, Manufacturing, and Control Information for Human Gene Therapy Investigational New Drug Applications," "Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus during Product Manufacture and Patient Follow-up," and "Long Term Follow-Up After Administration of Human Gene Therapy Products." Gottlieb noted, "Our goal is to help these innovations advance in a framework that assures the safety and effectiveness of these resulting treatments, and continues to build peoples' confidence in this novel area of medicine."