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Wednesday, October 31, 2018

Intellia Therapeutics announces results from ATTR studies


Intellia Therapeutics (NTLA) announced results from its transthyretin amyloidosis, or ATTR, non-human primate, or NHP, studies, conducted in collaboration with Regeneron (REGN), related to its enhancements of the cargo components of its lipid nanoparticle, or LNP-based delivery system. These novel component enhancements, which are part of the ongoing development of its proprietary and modular in vivo delivery platform, have produced unprecedented results, achieving up to 78% liver editing in our most recent NHP study. The corresponding transthyretin, or TTR, protein reduction at 21 days showed a decrease from baseline of up to 96% after a single dose. This substantially improved level of liver editing, achieved with a lower dose and well-tolerated safety profile, compares with mean editing levels of 34 percent in Intellia’s previously reported NHP studies. Based on these new data, the company is pursuing confirmatory studies with the goal of integrating enhanced cargo components in its investigational new drug, or IND-enabling studies and submission of an IND for ATTR. In addition, the company intends to apply these technology improvements to the rest of its in vivo product pipeline.

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