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Companies Expand Cell Therapy Collaboration To Pursue CRISPR/Cas9-based Genome Editing.

CAMBRIDGE, Mass., December 6, 2018 -- Intellia Therapeutics, Inc. (NTLA), which is developing curative therapeutics using CRISPR/Cas9 technology, said it is expanding its cell therapy collaboration with Novartis to include the ex vivo development of innovative cell therapies using certain ocular stem cells.

As part of the updated collaboration terms, Novartis will have the right to develop CRISPR/Cas9-based products for one or more targets using these stem cells.

Intellia will receive a one-time $10 million cash payment and, consistent with the original collaboration agreement, will be eligible to receive downstream success-based milestones and royalties.

Intellia will gain expanded rights to Novartis' lipid nanoparticle (LNP) technology for all genome editing applications in both in vivo and ex vivo settings.

This licensed LNP technology is the foundation of Intellia's proprietary modular delivery system of CRISPR/Cas9 for its in vivo product pipeline.

Intellia retains rights to all other in vivo and ex vivo applications of CRISPR/Cas9, including for eye disorders, subject to certain in vivo target selection options by Novartis set forth in the original agreement.

"Genome editing enhancements made by CRISPR/Cas9 will enable the next generation of cell therapies," said CEO John Leonard. "With Novartis we are broadening the ex vivo application of our CRISPR/Cas9 technology from hematopoietic stem cells, or HSCs, to ocular stem cells. Broader rights to Novartis' LNP technology will assist our efforts to apply this technology in ex vivo settings for the development of proprietary cell therapies, just as we have done to develop our proprietary modular delivery system for in vivo products in the liver and other organs."

Independent from its ex vivo collaboration with Novartis, Intellia also is advancing its wholly-owned ex vivo pipeline of immuno-oncology and autoimmune cell therapies.

Intellia's proprietary ex vivo programs include its acute myeloid leukemia (AML) program utilizing transgenic T cell receptors (TCRs) against Wilms' Tumor 1 (WT1), a target identified in collaboration with Ospedale San Raffaele (OSR).


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Title Annotation:Strategic Alliances
Publication:Stem Cell Research News
Date:Dec 17, 2018
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