CRISPR/Cas9-based gene editing holds promise across a range of gene therapy applications
Intellia Therapeutics has 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).
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.
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. 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," said Mr Nessan Bermingham, CEO and co-founder of Intellia.
