Gene therapy is saving lives, but key challenges to realize its full potential

It’s been a turbulent year in the world of gene therapy, with another negative safety signal when Novartis confirmed that two children treated with its gene therapy to treat spinal muscular atrophy, Zolgensma, had died of acute liver failure. On the positive side, there were three new approvals (Roctavian for severe hemophilia A, Zynteglo for beta thalassemia, and Skysona for cerebral adrenoleukodystrophy).