The first-ever US gene and cell therapy approvals of 2017 suggest that the industry stands on the brink of a fundamental shift in the way transformative therapies will be financed and accessed by patients. In quick order that acknowledged the clinical merits of the science, the FDA approved the first directly administered gene therapy, Luxturna (voretigene neparvovec-rzyl), as well as the first two CAR-T therapies, Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel).
What makes these therapies noteworthy is that we can call them potentially curative. They’re administered just once, or over a...