Commercialization of modern-day gene therapies is now a reality. Next-generation modalities such as chimeric antigen receptor T-cell (CAR-T) therapies are fully in launch mode in the US, where Novartis AG and Gilead Sciences Inc. are banking on the success of their one-time hematological cancer treatments Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel), respectively. Final approval of those CAR-T therapies has also occurred in the EU, with funding arrangements in place in the UK. In addition, the first in vivo gene therapy for an inherited disease is now available in the US, by way of Spark Therapeutics Inc.'s Luxturna (voretigene neparvovec - rzyl), and the EMA is currently evaluating what could be the next approval in the market, bluebird bio’s LentiGlobin (lentiviral beta-globin gene transfer) for transfusion-dependent beta thalassemia.
Presently, there are 25 unique gene therapies that have reached Phase III or have been filed for approval. Approximately 33...