Advances in cellular and gene therapies (CGTs) – referred to as advanced therapy medicinal products (ATMPs) in the EU – offer opportunities for treating and potentially curing diseases for which no other treatment options were previously available. This article examines three areas of these groundbreaking technologies: gene therapies and the search for safe and optimized vectors in genome editing; cellular therapies and their programmable synthetic gene circuitries; and organoids for tissue-engineered therapies and as possible personalized companion diagnostics. The article also addresses current guidance on ATMPs and CGTs from the European Medicines Agency (EMA) and US Food and Drug Administration (FDA), respectively, and the steps they have taken to deliver these new therapies to patients safely and speedily…
