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January 7, 2015

Novartis Launches CRISPR Collaborations with Intellia, Caribou

  • Novartis said today it is launching collaborations with a pair of partners that will enable it to use CRISPR genome editing technology to discover and develop new medicines. The value of both collaborations was undisclosed.

    The pharma giant will team up with Intellia Therapeutics to explore options for using CRISPR to engineer chimeric antigen receptor (CAR) T cells and hematopoietic stem cells.

    Both potential uses tie in with Novartis R&D priorities that the company has sought to advance in recent months.

    In October, Novartis disclosed preliminary study results showing that 27 of 30 pediatric and adult patients with relapsed/refractory acute lymphoblastic leukemia (90%) experienced complete remissions with CTL019, a personalized cellular immunotherapy being co-developed by the company with the University of Pennsylvania under an exclusive global research and licensing agreement signed in 2012.

    And in August, Novartis signaled again its interest in stem cell medicine by investing $35 million to take a 15% stake in Gamida Cell, developer of the investigational stem cell therapy NiCord.® The experimental drug—now in Phase I/II trials for hematological malignancies such as leukemia and lymphoma—received orphan drug designations by both the FDA and the European Medicines Agency, the company said yesterday.

    Novartis has an option to fully acquire Gamida Cell for $165 million. But that option is only available for an undisclosed limited time, and tied to achieving unspecified milestones related to the development of NiCord —milestones that Gamida Cell has said it anticipates being met this year. The deal came less than a year after the September 2013 launch of Novartis’ collaboration with Regenerex allowing the pharma to use the latter’s hematopoietic stem cell-based Facilitating Cell Therapy (FCRx) platform, which has been investigated in kidney transplantation.

    Under its collaboration with Intellia, Novartis will receive exclusive rights to develop all collaboration programs focused on engineered CAR Ts, as well as the right to develop an undisclosed number of targets for ex vivo editing of HSCs. In addition, Novartis is receiving nonexclusive rights for limited in vivo therapeutic applications of CRISPR systems.

    Novartis also agreed to increase an equity investment made earlier in Intellia through an upfront payment of undisclosed amount, and consented as well to provide technology access fees and funding for R&D programs during the five-year term of the collaboration. Intellia could also receive downstream payments tied to successfully achieving milestones, as well as royalties.

    Intellia’s launch was announced November 18. The startup received an undisclosed amount of financing from Novartis, as well as $15 million each from Atlas Venture and Caribou Biosciences, in a Series A financing round.

    Caribou is the second company with which Novartis is launching a CRISPR-related collaboration. The pharma giant said it plans to use Caribou’s foundational CRISPR platform and intellectual property as a research tool for drug discovery. Novartis is receiving nonexclusive rights to Caribou's CRISPR platform for research conducted during the collaboration, and has agreed to fund the companies’ one-year research program. Novartis is also making a series A equity investment of undisclosed amount in the company.

    Caribou's CRISPR technology is based on research by the laboratory of Jennifer Doudna, Ph.D., of the University of California, Berkeley, and collaborators. Caribou'sCRISPR technologies IP portfolio is centered on an exclusive license to the foundational CRISPR-Cas9 work from the University of California and the University of Vienna.

    The Novartis-Caribou partnership is designed to draw upon Intellia's CRISPR expertise as well as Novartis’ expertise in research and in cell and gene therapy development at the Novartis Institutes for BioMedical Research (NIBR).

    “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,” NIBR President Mark Fishman said in a statement. “Much remains to be learned, and we are delighted to explore these directions with colleagues from Intellia and Caribou.”

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