This week marked a the first time the FDA has approved a gene therapy treatment for patients in the US. The therapy, Kymriah, is aimed at pediatric and young adult patients with a form of B-cell precursor acute lymphoblastic leukemia. The treatment includes collecting T-cells from the patient and modifying them with a lentiviral vector into chimeric antigen receptor (CAR) T cells, specifically making the immune cells able to target the cancer cells. While this is the first gene therapy drug to be approved by the FDA, there are currently over 250 in the clinical trials pipeline according to ClinicalTrials.gov. Hopefully this is the first in a number of novel and efficacious new therapies which will come out in the coming years. Additionally, as technology continues to evolve, costs will also hopefully continue to drop making such treatments more available worldwide. Are you working on any similar therapies?