Despite the great potential of gene therapies, only a few drugs have received market approval from regulators. To effectively bring next-gen gene therapies from bench to bedside, there is a strong need for alternative testing platforms that recapitulate human pathophysiology and help making accurate predictions.
Human induced pluripotent stem cells (iPSC)-derived models are a promising alternative to support and supplement animal testing. They recapitulate disease hallmarks in vitro and can be integrated in discovery to facilitate the selection of the most promising candidates, saving resources and reducing the translational gap.
