Intellia delivers transformative potential with in vivo gene editing 

Intellia and partner Regeneron have generated the first clinical data indicating CRISPR-based gene editing may live up to its promise of creating one-time, curative therapies for systemic diseases — a breakthrough that represents a looming threat to other new modalities.

The data, though only from six patients, provide the first hint that CRISPR-based therapies could displace other new modalities in indications where those technologies are just finding a footing on the market. Still, critical questions of durability, off-target editing and delivery beyond the liver need to be answered to assess the reach of the technology’s disruptive potential, and how quickly it can get there...