8:53 AM
Mar 01, 2018
 |  BC Innovations  |  Translation in Brief

Intellia’s delivery address

How Intellia is testing the limits of its LNP delivery system

Intellia Therapeutics Inc. (NASDAQ:NTLA) published data supporting its lead in vivo gene editing program and providing proof of concept for its delivery technology this week. Now, the company wants to see just how far the delivery platform extends.

In Cell Reports, Intellia researchers published a collection of data presented at scientific conferences over the past year showing delivery of CRISPR-Cas9 (CRISPR-associated protein 9)-based therapies using a lipid nanoparticle (LNP) delivery vehicle can help treat transthyretin amyloidosis in mice.

The data, plus ongoing non-human primate (NHP) studies, will support the company’s IND submission planned for next year, but also bring up questions about the delivery technology that Intellia plans to address in its preclinical research.

According to SVP of Innovative Sciences Thomas Barnes, the company is now addressing questions that include how long the Cas9 and guide RNA (gRNA) need to persist in patient cells, whether LNPs can be targeted to non-liver tissues and how the technology should be tweaked...

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