Ex vivo and in vivo genome editing: a regulatory scientific framework from early development to clinical implementation

Recent advances in human genome science have paved the way to a new class of human gene therapies based on gene editing, with the potential to provide a long-lasting curative strategy for many debilitating and complex disorders, for which there is an unmet medical need. Therapeutic genome editing encompasses both ex vivo and in vivo gene correction modalities, for which similar and also application-specific considerations apply, which dictate the overall strategy to be followed from a scientific, clinical and regulatory perspective. Here, the major regulatory barriers to successful clinical implementation are discussed, together with the key issues to be considered for generating safe (minimizing risks of tumorigenesis and off-target effects) and effective gene editing-based medicines for application in regenerative medicine.