Genetics

Intellia Therapeutics (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology, presented data from studies demonstrating a decrease in amyloid deposition in multiple tissues of a humanized mouse model of hereditary transthyretin amyloidosis (ATTR) after a single dose of lipid nanoparticles (LNPs). As quoted in the press release: The company …

Intellia Therapeutics (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology, presented data from studies demonstrating a decrease in amyloid deposition in multiple tissues of a humanized mouse model of hereditary transthyretin amyloidosis (ATTR) after a single dose of lipid nanoparticles (LNPs).

As quoted in the press release:

The company also presented non-human primate (NHP) data from initial studies showing a therapeutically meaningful level of TTR protein reduction that correlated with robust and significant liver editing following a single administration of LNPs. Yong Chang, Ph.D., vice president, safety pharmacology, Intellia, presented these data today, at Cold Spring Harbor Laboratory’s (CSHL) fourth meeting on Genome Engineering: The CRISPR-Cas Revolution, in Cold Spring Harbor, N.Y.

“As a follow-up to our rodent and NHP data released previously, we shared data on the extensive preclinical characterization of our therapeutic candidates. Our leads, used in conjunction with our modular LNP delivery system, result in reductions in TTR protein levels in NHPs that, when achieved in humans, are associated with therapeutic benefit,” said Intellia President and Chief Executive Officer John Leonard, M.D. “Now, with IND-enabling activities for our lead in vivo program, ATTR, underway, we are one step closer to realizing the potential of CRISPR/Cas9 genome editing for developing curative treatments for chronic genetic diseases like ATTR. These additional activities are focused on confirming these initial results and identifying appropriate dose ranges for the development of potential human therapeutics.”

Click here to read the full press release.

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