Marketing application for new ADA-SCID gene therapy submitted to EMA

GSK (GlaxoSmithKline), Fondazione Telethon (Telethon) and Ospedale San Raffaele (OSR) recently announced the submission of a marketing application to the European Medicines Agency (EMA) for a gene therapy (GSK2696273) to treat patients with adenosine deaminase severe combined immunodeficiency syndrome (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available. For more information see the official press release here.