Novartis has already invested in Intellia Therapeutics. Now it wants to use Intellia’s cutting-edge gene-editing tools on one of the world’s most promising new kinds of medicine.
In a deal that brings together two of the highest-profile fields in biotech, healthcare giant Novartis (NYSE: NVS) has negotiated with tiny Intellia for exclusive rights to the Cambridge, MA-based start-up’s gene-editing tools known as CRISPR/Cas9. It’s the first deal of its kind featuring the nascent gene-modification technology that has become an indispensable tool in bioresearch labs.
When mulling how to move its technology into therapeutics, Intellia weighed a big fundraise and go-it-alone strategy against an early partnership. CEO Nessan Bermingham (pictured) said the choice was to “leverage another group’s capabilities and maximize our opportunities to get into the clinic and onto the market.”
Novartis has clinical experience with cell therapy as well as a manufacturing facility in place.
In a statement, Novartis Institutes for BioMedical Research president Mark Fishman said, “We have glimpsed the power of CRISPR tools in our scientific programs in NIBR, and it is now time to explore how to safely extend this powerful technology to the clinic.”
Intellia isn’t the only company developing CRISPR/Cas9 technology. Editas Medicine, also of Cambridge, and London, UK-based Crispr Therapeutics are two others racing to turn the research-lab phenomenon into new therapeutics. But who owns which pieces of this potential Nobel-winning biotech discovery is a matter of debate—and legal tussle.
This is the first CRISPR/Cas9 biotech-pharma deal, but other gene-editing technologies that use a similar concept—enzymes, which are nature’s molecular scissors, guided to snip just the right spot in the DNA—have made big development strides, attracted big pharma partners, or both.
For example, Sangamo BioSciences (NASDAQ: SGMO) of Richmond, CA, has taken its zinc finger technology into the clinic to fight HIV and has partnered other preclinical programs with Biogen Idec (NASDAQ: BIIB) and Shire (NASDAQ: SHPG).
Novartis will use CRISPR/Cas9 in its own immunotherapy program that uses chimeric antigen receptor T-cells, or CART. In recent months, CART programs have made the deadly hematological cancers of dozens of people disappear, including 16 of 30 kids with acute lymphoblastic leukemia in Novartis’s Phase 1 trial. (All the CART results are from early stage trials and need to be confirmed in larger patient groups; Novartis has moved its program into Phase 2.)
Novartis was an investor in Intellia’s $15 million Series A round. Now it has paid Intellia an undisclosed but significant sum of money—I’ll explain why it’s significant in a moment—to use Intellia’s suite of tools in its CART therapy, which requires a patient’s T cells to be genetically engineered outside the body, then reintroduced. It’s those T cells, modified to attack tumor cells with a certain protein on their surface, that have done such a great job so far eradicating people’s leukemias and lymphomas.
Novartis presumably plans to use CRISPR/Cas9 to modify the T cells, but those plans weren’t immediately clear. Intellia CEO Bermingham implied that CRISPR/Cas9 would allow cellular engineers to more broadly exploit the activity of T cells, which are the attack dogs of the immune system.
Novartis will also work together with Intellia to use CRISPR/Cas9 to go after diseases mediated by hematological stem cells (HSCs). HSC-mediated diseases include sickle-cell disease and beta thalassemia, but Bermingham declined to say which HSC-mediated diseases specifically the Intellia-Novartis partnership planned to address.
The specific dollar amounts in the deal were not disclosed, but Bermingham said the cash, combined with the $15 million Series A that closed last November, gave Intellia at least a five-year cushion without the need to tap more private funding. In that time, he said, Intellia should be able to move programs not licensed to Novartis into clinical trials. He declined to estimate clinical timelines more specifically.
The Novartis collaboration lasts five years. At various points during those five years, the two companies will stop and assess their joint development of HSC programs, potentially dividing their ownership between the two camps.
Intellia is not the originator of its own platform. It has exclusive license to CRISPR/Cas9 through Caribou Biosciences, a Berkeley, CA, startup. Caribou has cut its own deal with Novartis, also announced today: Novartis gets non-exclusive rights to use Caribou’s CRISPR/Cas9 technology for research, and Caribou gets a year of funding plus cash toward a Series A funding round. (Terms were not disclosed.)
Caribou’s IP stems from the University of California, Berkeley, where biochemist Jennifer Doudna worked on a crucial idea with others at Berkeley and beyond. They took a defense system that bacteria deploy against viruses, first discovered a quarter-century ago by Japanese researchers, and made key changes that turned the system into a gene-modification tool. They published their work in Science in 2012. Doudna’s group and researchers affiliated with Editas are so far the two main parties contesting each other’s IP, although it would be no surprise if the patent disputes become multi-sided.
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