How drug developers can step up to meet regulatory expectations for gene therapy chemistry manufacturing and controls.
After well publicized stumbling blocks in the late-90s, vigor was restored to the gene therapy field in 2017 when the U.S. FDA greenlighted two chimeric antigen receptor (CAR) T-cell treatments for acute lymphoblastic leukemia, followed by the first directly administered gene therapy, developed to treat a hereditary form of vision loss.
Years later, there are still only a handful of approvals in the space (two gene therapies and five CAR-T cell therapies), but pipelines are brimming with potential.
