Posted by Michael Wonder on 02 May 2019
FDA's efforts to advance the development of gene therapy
1 May 2019 - 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 haemophilia 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.
