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Intellia Therapeutics Demonstrates Pioneering CRISPR/Cas9 Genome Editing Efficiency Data Using Lipid Nanoparticle Delivery Technology

  • First to demonstrate single dose, in vivo results, showing approximately a 97 percent reduction in serum transthyretin protein levels
  • Durability data show stable liver editing for at least four months
  • Increased liver editing efficiency reported to date at 70 percent, following a single dose

CAMBRIDGE, Mass., March 08, 2017 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NTLA), a leading genome editing company focused on the development of potentially curative therapies, today reported updated data showing increased levels of genome editing efficiency in vivo and durability results with its CRISPR/Cas9 technology, following a single administration. Using its lipid nanoparticle (LNP) technology, Intellia achieved approximately a 97 percent reduction in serum transthyretin (TTR) protein driven by 70 percent gene editing efficiency in the mouse liver.

These results are extremely promising, as they demonstrate compelling activity with lipid nanoparticles observed in the liver following a single dose,” said Executive Vice President, R&D John Leonard, M.D. “We are excited by the extent of the effect, which confirms the power of CRISPR/Cas9 for potential therapeutic uses. These high levels of gene editing are the result of Intellia’s effort to identify highly efficient delivery of CRISPR/Cas9 using lipid nanoparticles. The data advance our efforts as we look to transform the way we treat disease.”

The data are being presented for the first time at the Cowen and Company 37th Annual Health Care Conference in Boston on Wednesday, March 8, 2017 at 8:40 am ET. Complete data are being presented on March 22, 2017 at the Le Stadium Conference on Messenger RNA Therapeutics in Orleans, France. Data showed robust editing and sustained results including:

  • Progress in achieving in vivo gene editing in the TTR locus, with an efficiency of approximately 70 percent in the...