Intellia presented data demonstrating decrease in amyloid deposition

Intellia Therapeutics last night presented data from studies “demonstrating a decrease in amyloid deposition in multiple tissues of a humanized mouse model of hereditary transthyretin amyloidosis after a single dose of lipid nanoparticles.” The company also presented non-human primate 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.” In one humanized mouse study, researchers found that editing rates were dose-responsive, and also observed liver editing up to 80% across multiple lead guide candidates, Intellia said in a press release. In a second humanized mouse study, researchers observed a lack of amyloid deposits in relevant tissues, including the stomach, colon, sciatic nerve and dorsal root ganglion, and therefore, a potential reversal in disease, the company added. “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 CEO John Leonard. “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.”

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