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Intellia Therapeutics, Novartis Form Collaboration to Develop New Cell Therapies Using CRISPR/Cas9 Technology

January 7, 2015

Five-Year Collaboration Focused on CART and Hematopoetic Stem Cell Applications across a Range of Therapeutic Areas

CAMBRIDGE, Mass.—January 7, 2015—Intellia Therapeutics, a leader in the development of therapeutic products using CRISPR/Cas9 technology for gene editing and repair, today announced a five-year research and development collaboration with Novartis to accelerate the ex vivo development of new CRISPR/Cas9-based therapies using chimeric antigen receptor T cells (CARTs) and hematopoetic stem cells (HSCs). This collaboration comes only three months after Intellia was launched by Atlas Venture and Caribou Biosciences, providing an important validation of Intellia's team and capabilities.

CRISPR/Cas9-based gene editing holds promise across a range of gene therapy applications, including blood disorders, cancer and other genetic based diseases. It has been shown to be an efficient and precise method for gene editing across multiple cell and tissue types, making it an ideal platform for ex vivo applications, such as CART- and HSC-based therapies, as well as in vivo applications.

Under the terms of the agreement, Novartis receives exclusive rights to develop all collaboration programs focused on engineered CARTs. Within HSCs, Novartis and Intellia will jointly advance multiple programs, and have agreed to a process for assigning development and ownership rights, which will enable Intellia to develop its own proprietary internal HSC pipeline.

In addition to increasing its equity holding in Intellia, Novartis is making an upfront payment, and providing technology access fees and funding for R&D programs during the five-year term of the collaboration. Intellia is also eligible to receive downstream success-based milestones and royalties. Intellia will gain access to certain Novartis intellectual property and technology for the development of its own product pipeline. Intellia also reserves the right to pursue additional enabling partnerships in other areas of therapeutic interest.

"Our collaboration with Novartis is an important building block for Intellia that will greatly accelerate our effort to translate the promise of CRISPR/Cas9 into meaningful advances for patients," said Nessan Bermingham, Ph.D., Chief Executive Officer and co-founder of Intellia. "CARTs and HSCs represent two of the most immediate opportunities for CRISPR therapeutic development, and Novartis, as a leader in this space, is the ideal partner with which to develop strong product pipelines in these areas."

About Intellia Therapeutics
Intellia Therapeutics was formed in 2014 to lead the industry in one of the most promising new areas of therapeutic development: gene editing and repair using CRISPR/Cas9 technology. Intellia holds exclusive access to one of the most comprehensive intellectual property platforms available for the therapeutic use of CRISPR/Cas9. Intellia closed a Series A round in 2014 led by Atlas Venture and Novartis.

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Jennifer Mound Smoter
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