Intellia: Balancing in vivo risk with validated targets

Intellia’s decision in 2014 to develop systemically delivered, in vivo gene editing therapies meant embracing some of the highest levels of technology risk ever encountered by a new technology platform. The biotech offset that risk by selecting targets already validated by other genetic medicine modalities in humans.

The company now appears to be on a path to clinical success, but the tradeoff of its target and indication selection strategy may be modest commercial opportunity...