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Clinical trials of genome-editing agents — including CRISPR–Cas9 editors, zinc finger nucleases and TALENs — are pushing ex vivo, immuno-oncology and in vivo treatment frontiers.
Gene-editing programmes have been slowly trickling into the clinic since 2005. But the pace is picking up. In the past year and a half alone, at least 11 such programmes entered the clinic in the US or EU. And 6 of these rely on CRISPR–Cas-based editors. With the clinical-stage pipeline now spanning ex vivo, immuno-oncology and in vivo applications, the genome-editing pipeline is taking form.