One of the few pioneers in the burgeoning gene editing field, Intellia $NTLA has directed attention to its IND-enabling animal data for some time. Now, it’s come out with a published scientific study — complete with a few “under the hood” highlights — to back the hype up.
The study, published in Cell Reports Tuesday, formally summarizes what Intellia has been touting since last March: the lipid nanoparticle technology it’s used to deliver CRISPR/Cas9 gene editing has proven to essentially eliminate all transthyretin (TTR) proteins in the liver of mice. The effects of one dose lasted for at least 12 months, suggesting that the gene editing happened not only in normal cells but the stem cell population of the hepatocytes.
While the report cites a 97% target protein knockdown, Intellia SVP of innovative sciences Thomas Barnes explained to me that was an average number across multiple cohorts. On a given day in a certain animal, the tech would likely be found to have achieved “quantitative editing” — effectively zero TTR.
Another highlight from the study was that the CRISPR/Cas9 material stayed in the body only “transiently,” lowering safety concerns. The safety of the adeno-associated virus (AAV) delivery vehicle that some other companies use has been called into question in recent weeks. Editas, one of Intellia’s main rivals, lost some momentum because of an AAV manufacturing issue.
“You don’t really want the Cas9 component hanging around forever, or even for a very long period of time. You want them to be there just long enough to do the edits that they need to do, and then you want it all to go away,” Barnes said. “And that’s essentially what we’ve shown our system can do.”
The publication comes as Intellia is knee-deep in non-human primate studies, which are scheduled to wrap up in the first part of the year. During the rodent study, researchers also did tests on rats which shed light on the relevant parameters for re-optimizing the tech in larger animals. That has played an instructional role in the current trials by arming researchers with knowledge of what areas to play and tweak.
With a new lipid that’s been designed to be scalable in commercial operations, Intellia is ready to brace an IND in 2019, though Barnes was careful not to specify a time just yet.
“This is a very important milestone in the path of turning LNP-based approaches in CRISPR into a truly clinic-ready agent,” he said.
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