On September 26, 2017 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on the development of potentially curative therapeutics using CRISPR/Cas9 technology, reported the full 12-month data of its completed long-term mouse study, demonstrating robust and durable in vivo genome editing post single-dose, intravenous administration using its proprietary lipid nanoparticle (LNP) delivery system (Press release, Intellia Therapeutics, SEP 26, 2017, View Source [SID1234520654]).

The data presented at the 13th Annual Meeting of the Oligonucleotide Therapeutics Society in Bordeaux, France, demonstrated robust editing and durability:

Achieved and maintained approximately 97 percent reduction in serum TTR protein levels through one-year, following a single dose
Attained approximately seventy percent editing at the target DNA site in the liver through one year
The transient nature of LNP delivery was confirmed with 99 percent clearance of mRNA within 10 hours and of sgRNA within 72 hours in the liver
The genome editing treatment was well tolerated with no adverse events during the course of the 12-month study
“These results show the long-term persistence of edited TTR genes in the mouse liver, indicating the potential for sustained efficacy and tolerability of our CRISPR/Cas9 platform as we move to higher species,” said David Morrissey, Ph.D., senior vice president, Platform and Delivery Technology. “These data validate Intellia’s approach as we remain on track with our non-human primate studies, which will permit us to designate our development candidate and perform IND-enabling activities in 2018 for the development of potential therapies in humans.”