A new contender enters the race for cystic fibrosis gene therapy

As a monogenic disorder, cystic fibrosis (CF) has long been viewed as an attractive candidate for a one-size-fits-all gene therapy approach. Combined with the fact that the most clinically relevant target cells are located within the human airway epithelium (HAE), which was once thought to be readily accessible through aerosolized vector delivery, CF appeared to be the ideal disease for targeted gene therapy development. However, except for two currently ongoing studies, all clinical trials for viral vector-mediated, lung-targeted CFTR gene therapy have been stopped in their tracks before phase 3.