Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products

Gene and cell therapy research recently reached a fundamental milestone toward the goal to deliver new medicines for orphan diseases. In 2016, the European Commission granted market approval to GlaxoSmithKline ( GSK ) for ex vivo hematopoietic stem cell ( HSC ) gene therapy for the treatment of adenosine deaminase ( ADA )‐deficient severe combined immunodeficiency ( SCID ), a very rare congenital disorder of the immune system. The new medicine,...