Jan 07, 2015
  • Collaborations give Novartis access to novel CRISPR technology platforms for the discovery and development of new medicines
  • Intellia Therapeutics collaboration to explore therapeutic options for using CRISPR to engineer chimeric antigen receptor T-cells and hematopoietic stem cells
  • Caribou Biosciences collaboration focused on using CRISPR as a research tool for drug discovery 

Basel, Switzerland, January 7, 2015 - Novartis announced today that it has signed collaboration and licensing agreements with Intellia Therapeutics for the discovery and development of new medicines using CRISPR genome editing technology and Caribou Biosciences for the development of drug discovery tools. Intellia and Caribou are two of the leading biotechnology companies developing this novel technology.

CRISPR, an acronym that stands for clustered regularly interspaced short palindromic repeats, is an approach that allows scientists to easily and precisely edit the genes of targeted cells. In a short period of time it has proven to be a powerful tool for creating very specific models of disease for use in drug discovery and has great potential for use as a therapeutic modality for treating disease at the genetic level by deleting, repairing or replacing the genes that cause disease.

"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," said Mark Fishman, President of the Novartis Institutes for BioMedical Research (NIBR). " CRISPR has the potential to open a new branch of medicine, editing the genome to cure disease. Much remains to be learned, and we are delighted to explore these directions with colleagues from Intellia and Caribou. "

The alliance with Intellia combines the resources, research expertise and cell and gene therapy leadership at the Novartis Institutes for BioMedical Research with Intellia's rapidly growing CRISPR expertise and organization. Research and development activities will focus on using CRISPR ex vivo for engineering chimeric antigen receptor T-cells (CARTs) and hematopoietic stem cells (HSCs). The collaboration and licensing agreement with Caribou is focused on using Caribou's foundational CRISPR platform and intellectual property as a research tool for drug discovery.

Under the terms of the agreement with Intellia, Novartis is receiving exclusive rights to develop all collaboration programs focused on engineered CARTs and the right to develop an undisclosed number of targets for ex vivo editing of HSCs. In addition Novartis receives non-exclusive rights for limited in vivo therapeutic applications of CRISPR systems. Novartis is increasing its equity investment in Intellia, is making an upfront payment and will provide technology access fees and funding for R&D programs during the 5-year term of the collaboration. Intellia is also eligible to receive downstream success-based milestones and royalties. Under the terms of the agreement with Caribou, Novartis is receiving non-exclusive rights to Caribou's CRISPR platform for research conducted during the collaboration and is providing funding for the one-year research program. Novartis is also making an equity investment in the company.

The foregoing release contains forward-looking statements that can be identified by words such as "commits," "to explore," "focused on," "potential," "may," "remains," "will," "eligible," or similar terms, or by express or implied discussions regarding potential marketing approvals for products developed through the collaborations with Intellia Therapeutics and Caribou Biosciences, or regarding potential future revenues from such products, the application of the CRISPR genome editing technology, or any other technologies developed through these collaborations. You should not place undue reliance on these statements. Such forward-looking statements are based on the current beliefs and expectations of management regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that any products or other technologies will arise from the collaborations with Intellia Therapeutics and Caribou Biosciences. Neither can there be any guarantee that any products arising from or developed through these collaborations will be submitted or approved for sale in any market, or at any particular time. Nor can there be any guarantee that any product or other technology arising from or developed through these collaborations will receive regulatory approval or be commercially successful in the future. In particular, management's expectations regarding the CRISPR genome editing technology, potential products and other technologies could be affected by, among other things, the uncertainties inherent in research and development, including unexpected clinical trial results and additional analysis of existing clinical data; unexpected regulatory actions or delays or government regulation generally; the company's ability to obtain or maintain proprietary intellectual property protection; general economic and industry conditions; global trends toward health care cost containment, including ongoing pricing pressures; unexpected manufacturing issues, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis
Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, eye care, cost-saving generic pharmaceuticals, preventive vaccines and over-the-counter products. Novartis is the only global company with leading positions in these areas. In 2013, the Group achieved net sales of USD 57.9 billion, while R&D throughout the Group amounted to approximately USD 9.9 billion (USD 9.6 billion excluding impairment and amortization charges). Novartis Group companies employ approximately 130,000 full-time-equivalent associates and sell products in more than 150 countries around the world. For more information, please visit http://www.novartis.com.

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