Genetics

Intellia Therapeutics (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.” As quoted in the press release: “These data show that our proprietary lipid nanoparticle technology achieves significant and …

Intellia Therapeutics (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.”

As quoted in the press release:

“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.”

Click here to read the full press release.

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