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

Abstract

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, named Strimvelis™, is an advanced therapy medicinal product ( ATMP) (Salmikangas et al, 2015) originally developed by the San Raffaele Telethon Institute for Gene Therapy ( SR‐Tiget), a joint venture between Telethon Foundation and San Raffaele Scientific Institute. This ATMP is the first ex vivo stem cell gene therapy to receive regulatory approval anywhere in the world. Strimvelis™ consists of a single infusion of autologous gene‐corrected HSC and is prepared from the patient's own bone marrow ( BM) HSCs, which are genetically modified using a gamma‐retroviral vector to insert a functional copy of the ADA gene.