HAE data clear Intellia’s path for liver knockout indications

Intellia does it again, showing for a second time that its in vivo CRISPR-Cas9 platform is capable of knocking down a clinically relevant target in the liver, while providing the first evidence that the knockdown leads to clinical benefit.

The results throw open the door for Intellia Therapeutics Inc. (NASDAQ:NTLA) to tackle nearly any indication with a relevant gene knockdown target in the liver, while laying the foundation for the gene editing technology’s next frontiers, which include gene insertion and delivery beyond the liver...