Gene Therapy’s First Out-and-Out Cure Is Here
May 9, 2016
(MIT Technology Review) – A treatment now pending approval in Europe will be the first commercial gene therapy to provide an outright cure for a deadly disease. The treatment is a landmark for gene-replacement technology, an idea that’s struggled for three decades to prove itself safe and practical. Called Strimvelis, and owned by drug giant GlaxoSmithKline, the treatment is for severe combined immune deficiency, a rare disease that leaves newborns with almost no defense against viruses, bacteria, or fungi and is sometimes called “bubble boy” disease after an American child whose short life inside a protective plastic shield was described in a 1976 movie.