Novartis invests in gene-editing with Intellia

Novartis has staked a claim to the hot topic of gene-editing using the CRSPR/Cas9 technology with an investment in recently-formed Intellia Therapeutics.

Massachusetts based Intellia - which was only formed a few months ago with start-up backing from Atlas Ventures - was formed specifically to accelerate the development of the CRISPR/Cas9 technology, which can be used to switch genes on or off and repair those that are faulty.

Novartis has thrown its weight behind the company with a five-year alliance, with terms including an upfront payment, equity stake and ongoing R&D funding. The two companies will concentrate on developing therapies using chimeric antigen receptor T cells (CARTs) and haematopoetic stem cells (HSCs).

The ex vivo approach will see HSCs extracted from patients, modified using the CRISP/Cas9 gene-editing technology and injected back into patients. At the moment the companies are not making any specific statements about the diseases they plan to focus on during the collaboration.

Using CRISPR/Cas9, an RNA guide molecule that locks onto a particular genetic sequence is attached to the Cas9 nuclease that cuts both strands of DNA at a specific location. Once the DNA helix is cut, the CRISPR-Cas9 construct can be used to harness and manipulate the usual cellular repair mechanisms for DNA and introduce a different gene sequence to the excised portion.

Intellia has licensed rights to intellectual property held by Caribou Biosciences, which was set up by University of Berkeley scientist and CRISPR/Cas9 pioneer Jennifer Doudna.

Doudna shared the 2014 Dr Paul Janssen Award for Biomedical Research with Emmanuelle Charpentier of the Hannover medical School and Helmholtz Centre for Infection Research in Germany for their work in the field.

There are already signs of jostling for position with regards to intellectual property in CRISPR/Cas9, however, with Charpentier associated with a rival company - CRISPR Therapeutics - which is based in Switzerland.

Complicating the picture, Feng Zhang of the MIT-Harvard Broad Institute was awarded a patent on the technology last year, while Doudna has another patent pending. Zhang and Doudna were co-founders of Editas Medicine, a start-up that has licensed rights to the MIT patent, but has Doudna severed ties with the company.

The legal wrangling over ownership will take years to play out, and in the meantime companies are pressing ahead with exploiting CRISPR/Cas9 both as a research tool and as a platform for therapeutics development.

Intellia chief executive Nessan Bermingham said in a statement that the deal 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."

For more on the patent claims to the CRISPR technology, read this MIT Technology Review article: Who Owns the Biggest Biotech Discovery of the Century? (external link)