Intellia Therapeutics Reports Financial Results For Third Quarter 2016

  • First company to report high in vivo editing data using CRISPR/Cas9 in animal models
  • Further validation of lipid nanoparticle delivery platform
  • Ended quarter with $290.6 million in cash and cash equivalents

CAMBRIDGE, Mass., Nov. 01, 2016 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on the development of potentially curative therapeutics using CRISPR/Cas9 technology, today reported results for the quarter ended September 30, 2016 and provided an update on recent highlights and upcoming events.

"We have demonstrated substantial progress in our research, including being the first company to present data showing high levels of  in vivo editing in animal models using systemic lipid nanoparticles to deliver CRISPR/Cas9 components," said Nessan Bermingham, Ph.D., Chief Executive Officer and Founder, Intellia Therapeutics. "We continue to make further enhancements and remain focused on advancing the development of CRISPR/Cas9-based therapeutics for patients with severe unmet medical needs."

Recent Highlights
  • Intellia presented preclinical data demonstrating in vivo gene editing using lipid nanoparticles (LNPs) to deliver CRISPR/Cas9. These data were presented at the Genome Engineering: The CRISPR/Cas Revolution meeting in Cold Spring Harbor, New York. In several preclinical studies, the data showed:
    • Progress in achieving in vivo editing, reporting an efficiency of approximately 60 percent in mouse liver at the transthyretin (TTR) target site after a single intravenous administration, which was consistent across different lobes of the liver. This resulted in an associated decrease in serum TTR protein levels of up to approximately 80 percent;
    • Dose-dependent editing by LNP delivery;
    • Undetectable Cas9 mRNA and guide RNA (gRNA) in the liver at 72 hours post administration; and
    • Repair patterns in mouse liver cells in vivo being best predicted in vitro by primary mouse liver cells rather than cell lines. 
  • Intellia presented four posters at the recent European Society for Gene and Cell Therapy Congress (ESGCT) in Florence, Italy. The data presentations included an update on the Company's in vivo delivery and DNA repair data and new methods for analyzing off-target activity. In its presentation on off-target analysis, Intellia described improved computational methods for readily identifying guide RNAs with zero to few off-target events, an essential step in developing CRISPR/Cas9-based therapeutics.

Third Quarter 2016 Financial Results

As of September 30, 2016, Intellia had $290.6 million in cash and cash equivalents. Net loss for the third quarter 2016 was $7.5 million, compared to $3.0 million in the same period in 2015.

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