Most clinical trials (87%) are based on ex vivo programs in which cells are harvested from patients, engineered and then re-infused into the patients. In vivo programs, in which the technologies are the therapeutic themselves, are still complex and present significant challenges, and are therefore currently not a popular choice yet.
Clinical trials assessing gene editing-based therapeutics
Unlike in vivo approaches, in ex vivo approaches, genome editing technologies are an enabler to design new therapeutics. It is easier to edit genes ex vivo in a laboratory and the risk of off-target editing is very limited, largely…
