Novartis has teamed up with Intellia Therapeutics, which focuses on gene editing and repair, to create treatments from the US biotech’s stem cell technology.
The deal aims to accelerate the ex vivo (‘out of the living’) development of new CRISPR/Cas9-based therapies using chimeric antigen receptor T cells (CARTs) and hematopoietic stem cells (HSCs).
This sees cells collected from patients and then modified for therapeutic purposes before being injected back into patients – the first part of this being the ex vivo approach.
An in vivo (‘into the living’) approach can then be used to correct genes found inside cells, with applications in ophthalmic, central nervous system, muscle, liver, anti-infective and other diseases such as cancer.
This collaboration comes just three months after Intellia was launched by Atlas Venture and Caribou Biosciences, who both gave $15m to the start-up as well as an initial financial backing from Novartis who had not, until now, disclosed whether it would be looking for an extended R&D deal.
The biotech has seemingly garnered much attention from the industry in a short period, which is helped by the former chief scientific officer of AbbVie John Leonard being one of its new leaders.
The firm is still at a very early stage and is actively seeking to recruit an initial 30 people to its site in Cambridge, Massachusetts. Its technology is seen as the future of research across many diseases areas, meaning Novartis is looking to play the long game with this research deal.
Under the terms of the agreement, Novartis will receive exclusive rights to develop all collaboration programmes focused on engineered CARTs.
Within HSCs, Novartis and Intellia will together run a number of programmes and has 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, which the firm announced in November last year, Novartis is making an upfront payment and providing technology access fees and funding for R&D programmes during the five-year term of the collaboration. The amount of money involved has not been disclosed.
Intellia is also eligible to receive success-based milestones and royalties. Intellia will gain access to certain Novartis intellectual property and technology for the development of its own product pipeline.
The biotech will also reserve the right to pursue additional enabling partnerships in other areas of therapeutic interest, it says in a statement.
“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, chief executive and co-founder of Intellia.
He added: “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.”