When Moderna CEO Stephane Bancel said he wanted to buy into gene editing, the chatter over the potential target flared to a roar.
So you know we had to ask when we had Intellia CEO John Leonard, M.D., on the line. He, of course, laughed and didn’t say whether he’d pick up Bancel’s call.
“The plan right now is for the company to be independent,” Leonard said during Fierce Biotech’s Cell & Gene Therapy Summit.
Instead, Intellia wants to see just what its CRISPR/Cas9 gene editing technology can do before making major decisions about the company’s future as an independent biotech.
“We’re just in the early innings of this game,” Leonard said. “It’s an approach to see what the technology has to give.”
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The technology has, for the record, already been giving. Intellia broke new ground in June with early-stage first-in-human data for a gene editing therapy called NTLA-2001, which bested existing standard-of-care treatments for the rare disease transthyretin (ATTR) amyloidosis.
The Cambridge, Massachusetts-based biotech also recently received clearance from the FDA for human testing of NTLA-5001, an ex vivo CRISPR genome-editing cancer candidate, as well as an in vivo hereditary angioedema treatment called NTLA-2002.
But Leonard is not ruling out potential deals that could bring new technology into the Intellia fold—especially anything that can improve on delivery mechanisms in gene editing.
“We’re always looking for technology that can be brought to bear to genome editing,” he said. “We think we know what needs to be done once you get inside a cell, and, if there’s a particular gene or set of genes that need to be manipulated, we think we can do that as well as anybody on Earth, as far as we know.
“But delivery is something that can augment the work that we do. If people have technologies that can add to that, we’d always be interested in learning about that.”
Leonard continued, “We can’t be experts in everything. So we try to emphasize genome editing itself and look for ways to collaborate with others.”
Intellia has struck a number of different types of deals over the past few months, including a recent 10% stake in SparingVision, which marked another foray into ocular diseases. Leonard noted two deals signed in June, which included the launch of a new company to go after CAR-T cell therapies.
He also oversaw a buyback of rights to certain assets from Novartis that came from an earlier collaboration between the two companies. The companies have a research partnership in ex vivo sickle cell disease therapies and other undisclosed targets.
“It’s not so hard to imagine other circumstances where we have a technology and somebody adds a piece to it and we can find a way to progress the work,” Leonard said.
The Novartis deal isn’t Intellia’s only Big Pharma partnership. The ATTR amyloidosis therapy NTLA-2001 is being developed in a partnership with Regeneron that began in 2016. The companies also have an agreement in hemophilia A and B, and other pre-clinical research underway. Leonard said Regeneron has provided expertise in clinical research and adeno-associated viruses for delivery of gene therapies.
“It’s a nice example where we have certain strengths and they have other skills and we bring them together into something that neither of us could do alone,” the CEO said.
The NTLA-2001 results helped Intellia boost its stock price and market cap, which currently sits at $9.7 billion—a hefty sum for a clinical-stage biotech that is nowhere near commercialization. That position could certainly help Leonard and his business development team the next time they discover some interesting tech.