GSK EU approval for gene therapy18 Apr 2016 11:44
The world's first life-saving gene therapy for children, developed by Italian scientists and GlaxoSmithKline, has been recommended for approval in Europe, boosting the pioneering technology to fix faulty genes. The European Medicines Agency (EMA) said on Friday it had endorsed the therapy, called Strimvelis, for a tiny number of children with ADA Severe Combined Immune Deficiency (ADA-SCID) for whom no matching bone marrow donor is available. This could be the start of something big. Do not be put off by the fact that this is a niche disease with very few patients. It is the proof of principle here that is vital and this approval is a potential game changer