Gene Therapy Is Curing Hemophilia
June 13, 2016
(MIT Technology Review) – In April Maurits, an engineering designer, joined a study in which he was dosed with viruses packed with a correct version of the gene that codes for factor IX. Today at the European Hematology Association’s meeting in Copenhagen, the Philadelphia company that ran the gene-therapy study, Spark Therapeutics, is presenting results on four patients, him included. In all four, factor IX activity has reached about 30 percent of average. That’s enough to prevent bleeding when you get hit by a baseball or twist your ankle. It’s also been enough to let Maurits go without factor IX replacements since April. “There’s no other explanation than ‘It worked,’” says Maurits.