Novartis has signed collaboration and licencing 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 programmes 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 programmes 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 programmes 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 programme. Novartis is also making an equity investment in the company.