Gene therapy has been on the horizon for several decades and has now become a reality in the United States. There are now three approved products: two cell-based gene therapies for cancers of the blood (Kymriah and Yescarta) and one directly-administered gene therapy (Luxturna) for an inherited disorder of the retina of the eye. Numerous gene therapy products are in development to address unmet medical needs, such as those for the treatment of diseases ranging from hemophilia to spinal muscular atrophy. The progress and demand in this field is also evident in the more than 800 active investigational new drug applications for gene therapies on file with FDA.
To keep pace with the remarkable and continuing growth of gene therapy, FDA’s Center for Biologics Evaluation and Research (CBER) has also been growing to take on the challenges of product development and to work with developers to help address those challenges. The National Institutes of Health (NIH) has been and continues to be a leader in the field of gene therapy. Over the years, FDA has developed a comparable cadre of experts, who both review gene therapy applications and conduct applied research relevant to the field. FDA has made it a priority to field a strong bench of highly-skilled and knowledgeable experts in the cutting-edge science behind gene therapy, including genome editing technologies, novel gene delivery methods, and advanced manufacturing technologies.
Like millions of other Americans, Victoria Gray has been sheltering at home with her children as the U.S. struggles through a deadly pandemic, and as protests over police violence have erupted across the country. But Gray is not like any other American. She’s the first