Intellia Therapeutics Announces Publication in Cell Reports of Preclinical Data Demonstrating Effective CRISPR/Cas9 Genome Editing Using Lipid Nanoparticle (LNP) Delivery Technology

  • Intellia researchers achieved 97 percent reduction in serum transthyretin protein levels in mice through a single administration, with a durability of at least 12 months
  • CRISPR/Cas9 administered via LNP was well-tolerated with no observed adverse events
  • CRISPR/Cas9 components shown to be undetectable three days after administration

CAMBRIDGE, MA, USA I February 27, 2018 I Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company developing curative therapeutics using CRISPR/Cas9 technology, announced that Cell Reports will publish at noon ET today its manuscript, “A single administration of CRISPR/Cas9 lipid nanoparticles achieves robust and persistent in vivo genome editing.”

The lipid nanoparticle (LNP) delivery of Cas9 mRNA and sgRNA resulted in 97 percent reduction in mouse transthyretin (TTR) protein levels in the liver, and the reduction was sustained for at least 12 months. The publication also documents that CRISPR/Cas9 components were undetectable in mice within three days after administration of Intellia’s LNP delivery system. Researchers further demonstrated that Intellia’s LNP technology is a similarly robust and effective delivery method for CRISPR/Cas9-mediated knockdown in rats, a higher rodent species.

“These data show that our proprietary lipid nanoparticle technology achieves significant and enduring editing of the TTR gene through a single dose,” said David Morrissey, Ph.D., senior vice president, Platform and Delivery Technology, at Intellia. “Our lipid nanoparticle system is a transient expression system that enables CRISPR/Cas9 to make the intended gene edit and then clear from the cells. Minimizing the duration of CRISPR/Cas9 components in cells is desirable, as that may reduce the potential for safety issues associated with the continued presence of those components. The LNPs also allow us to re-dose, if needed, to attain the desired target effect. This paper details the most effective systemic delivery of CRISPR/Cas9 components reported to date, further supporting our IND-enabling activities this year and future potential treatments for liver-based genetic diseases.”

The data included in this publication build on earlier findings initially released last year at the Le Stadium Conference on Messenger RNA Therapeutics, and later presented at the 20th Annual Meeting of the American Society of Gene and Cell Therapy and the 13th Annual Meeting of the Oligonucleotide Therapeutics Society.

About Intellia’s transthyretin amyloidosis (ATTR) program

Transthyretin amyloidosis (ATTR) is a slowly progressive and debilitating disease caused by one of approximately 136 different inherited mutations in the TTR gene. Abnormal protein deposits caused by one of these genetic mutations may affect both the peripheral and autonomic nervous systems, resulting in a variety of symptoms that develop in people as early as age 20. (Sources: Amyloidosis Foundation and National Institutes of Health)

Intellia’s sentinel in vivo programs focus on the use of lipid nanoparticles (LNPs) for delivery of CRISPR/Cas9 components to the liver. The company’s lead in vivo program targets ATTR and is being co-developed with Regeneron Pharmaceuticals, Inc. Intellia aims to achieve knockout editing of specific DNA in mutated TTR genes within hepatocytes that cause damaging transthyretin protein deposits in heart, nerves and other tissues in the body. Non-human primate studies are ongoing and are anticipated to lead to IND-enabling activities in 2018.

About Intellia Therapeutics

Intellia Therapeutics is a leading genome editing company focused on the development of proprietary curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. Our combination of deep scientific, technical and clinical development experience, along with our leading intellectual property portfolio, puts us in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com; follow us on Twitter @intelliatweets.

SOURCE: Intellia Therapeutics

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