Gene Therapy Comes of Age
Two breakthroughs this year made it possible to talk about gene therapy coming of age, and within a generation, envision its wide use in reversing disease. One was the European Union’s November 2 approval of uniQure’s marketing authorization application for Glybera® (alipogene tiparvovec) for lipoprotein lipase deficiency, the Western world’s first approved gene therapy drug.
“The precedent of an approved AAV [adeno-associated virus] product is going to help enormously in the further business development and commercialization of gene therapy,” said James M. Wilson, M.D., Ph.D., of the University of Pennsylvania Perelman School of Medicine, editor-in-chief of Human Gene Therapy, HGT Methods, and HGT Clinical Development, all published by GEN Publisher Mary Ann Liebert, Inc.
The other milestone was the success by researchers at University of Pennsylvania’s Perelman School of Medicine of a gene therapy that used infusions of patients’ own T cells genetically engineered to attack their tumors. The technique showed nine of 12 leukemia patients in remission, two of them for more than two years.