Intellia’s CEO Has Cambridge-Based Company Moving Forward on Positive in vivo Data

March 20, 2017
By Alex Keown, BioSpace.com Breaking News Staff

CAMBRIDGE, Mass. – As 2017 rolls onward, Intellia Therapeutics is finding itself in a good position regarding its pipeline. The company continues to build upon 2016 successes, including positive preclinical in vivo data for its CRISPR-Cas9 delivered lipid nanoparticle (LNP) technology.

Earlier this month, the company released updated data showing high levels of genome editing efficiency in vivo and durability results with its CRISPR-Cas9 technology, following a single administration in mouse models. Using its LNP technology, Intellia achieved approximately a 97 percent reduction in serum transthyretin (TTR) protein driven by 70 percent gene editing efficiency in the liver, the company said in its announcement. In the study, Cas9 mRNA was co-formulated with chemically synthesized gRNAs targeting the mouse TTR gene, and administered via one intravenous tail vein injection, according to Intellia.

Nessan Bermingham, chief executive of Intellia Therapeutics, told BioSpace in an exclusive interview that the results were incredibly promising when it comes to potential treatments of the liver through CRISPR-Cas9 technology. Cas9 is an enzyme that can be programmed with RNA to cut DNA at specific, targeted locations within the genome, which allows for easier, more specific gene editing.

“When we think of a therapeutic standpoint to treat someone once and have this effect, that’s unprecedented today. As far as we know, this is the first data showing such a significant delivery,” Bermingham said.

Now the company plans to take the process to the next level with primates.

It wasn’t just the in vivo data that had Bermingham excited. He said the company has data from multiple studies that have great potential. Intellia is also seeing positive results in studies in hepatitis B. With about 240 million people affected by HBV, Bermingham said genome editing provides a goal of being able to provide a real cure for the disease.

Intellia is also working on a Transthyretin Amyloidosis (ATTR) program being developed with Regeneron. The six-year agreement was struck in April 2016. Under terms of the agreement, Regeneron has rights to discover and develop CRISPR-based products against up to 10 targets, focused primarily on therapies for a broad range of diseases that may be treated by editing genes in the liver. Of the 10 targets, Regeneron can select up to five non-liver targets.

Intellia also has an ex vivo program underway to use chimeric antigen receptor T Cells (CAR-T) and hematopoietic stem cells. Intellia struck a deal with Novartis in January 2015 to focus on CRISPR-Cas9 applications with CART and HSC-based therapies.

One of the benefits of CRISPR-Cas9 that Bermingham touted was the short time the editing effects remain in the body. When using the technology, Bermingham said it’s important that the technology does what it’s designed to do, then goes away. Current data shows that after 72 hours of the treatment, they cannot detect the CRISPR-Cas9.

“You should want it in to make the edit and then be gone, a seamless treatment,” Bermingham said.

Even as the company moves forward with its research, there is an ongoing debate about the patents surrounding CRISPR-Cas9 technology. In February, the U.S. Patent Office ruled the Broad Institute of Harvard University and the Massachusetts Institute of Technology were the owners of the technology. That decision is likely to be appealed by other players in the CRISPR game, including the University of California.

While legal battles surrounding the CRISPR-Cas9 patents continue, Bermingham said the day-to-day research at Intellia has not been impacted. He said the company’s R&D and its partnerships are continuing in a normal manner. He said the Patent Court decision is the first stage in a multi-stage process.

“There are other avenues for things going forward,” Bermingham said. “This is not the end, it will continue to progress. This is just another phase in… what is a long road resolving IP conflicts.”

Intellia closed out 2016 by moving into a larger office space, something that Bermingham said is still amazing to see daily. When the company launched in 2014 there were only four employees in a small space. But now, they’ve grown rapidly in only a few years.

“Here we are at the end of the year moving into a 65,000 square foot facility,” Bermingham said.

He said the company is looking to develop a therapeutic approach that is unprecedented.

“When you think about it, we’re talking about a single treatment to cure them (patients) of a disease. This can address the driver, the fundamental cause of a disease,” he said. “In due course, this technology will allow us to really talk about personalized medicines. We haven’t had the tools to talk about that from a serious standpoint.”