Novartis Licensing Biotechs’ Gene-Editing Technologies

(NIST.gov)

7 January 2015. Pharmaceutical maker Novartis is licensing technologies from two biotechnology companies that enable the editing of human genomes to cure disease. Financial details of the agreements with Caribou Biosciences Inc. in Berkeley, California and Intellia Therapeutics in Cambridge, Massachusetts were not disclosed, but involve equity investments, initial payments and research support, and milestone payments from Novartis. Caribou is a founding partner and Novartis is already an investor in Intellia.

The deals with Caribou and Intellia involve a technology known as clustered regularly interspaced short palindromic repeats or CRISPR, that harnesses an enzyme called Cas9 to edit genes in targeted cells. CRISPR directs Cas9, guided by RNA produced by the genome, to disease-causing mutations in the genome to deactivate or replace the problem genes. The companies say the processes can be designed to edit multiple genes at the same time, as well as work with other proteins to control gene expression.

Caribou Biosciences was founded in 2011 and licenses research on CRISPR in the UC-Berkeley lab of Jennifer Doudna, a co-founder of the company, for drug discovery and screening. The company says its technology provides a more stable platform for identifying therapeutic targets than current techniques. In the deal, Novartis, based in Switzerland, will fund a one-year research project with Caribou and gain a non-exclusive license to Caribou’s CRISPR-Cas9 technology. Novartis is also making an equity investment in Caribou.

Intellia, founded in November 2014 by Caribou and Atlas Venture, an early-stage life sciences venture capital company, develops and commercializes CRISPR-Cas9 therapies. With Novartis, Intellia plans to harness CRISPR to engineer chimeric antigen receptor T-cells, immune-system cells that attract antigens producing antibodies, cultured and grown in the body, then infused back into patients to attack disease-causing cells. The Novartis collaboration also covers Intellia’s work with hematopoietic or blood-forming stem cells, which they plan as well to culture and grow in the lab for transplant in patients.

In the five-year deal with Intellia, Novartis receives an exclusive license to develop results of the collaboration on engineered chimeric antigen receptor T-cells. Novartis also can propose an undisclosed number of targets for hematopoietic or stem cells, as well as a receive non-exclusive license to CRISPR-Cas9 therapies designed to work inside the body, rather than grown in the lab and transplanted.

Intellia is receiving an initial payment from Novartis, as well as funding support for its research. In addition, Intellia will be eligible for staged milestone payments and subsequent royalties on sales. Novartis is also making another equity investment in Intellia.

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