Gene therapy takes step forward as GSK files for EU approval
LONDON – GlaxoSmithKline said on Tuesday it had submitted a gene therapy for approval in Europe, becoming the first big drugmaker to seek marketing authorization for the technology to fix faulty genes.
Reuters reported last week that the move was imminent. It marks the latest sign of a renaissance in gene therapy after some disastrous clinical trial results in the late 1990s and early 2000s.
GSK’s product, developed with Italian scientists, is designed to treat a tiny number of children with ADA Severe Combined Immune Deficiency (ADA-SCID) for whom no suitable bone marrow donor can be found. – Reuters