Intellia CRISPR tech leads to 97% TTR knockout

Intellia Therapeutics Inc. (NASDAQ:NTLA) gained $0.53 to $13.56 on Wednesday after it said a single administration of its CRISPR-Cas9 technology, delivered via lipid nanoparticles in a mouse model, led to a 97% reduction in transthyretin serum protein levels. The company said it was the first to produce such a response in vivo.

Intellia said the highest test dose of its gene editing approach, which uses the delivery system to knock out the TTR gene in hepatocytes, was effective in 70% of liver cells. TTR serum reductions were sustained through four months, with one year of follow-up planned. The company also said Cas9 mRNA and guide RNA were cleared quickly, and were undetectable after 72 hours...