Novartis to use gene editing tool with CAR-T therapies
This article was originally published in Scrip
Novartis has signed collaboration and licensing agreements with Intellia Therapeutics for the discovery and development of new medicines using the CRISPR genome editing technology. It simultaneously entered a deal with Caribou Biosciences for the development of drug discovery tools in the nascent field.
"CRISPR has the potential to open a new branch of medicine, editing the genome to cure disease," stated Mark Fishman, president of the Novartis Institutes for BioMedical Research (NIBR).
CRISPR (clustered regularly interspaced short palindromic repeats) is an approach that allows scientists to edit the genes of targeted cells. Novartis says that 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. Novartis believes the technology has great potential 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 programs in NIBR, and it is now time to explore how to safely extend this powerful technology to the clinic," added Dr Fishman. "CRISPR has the potential to
Novartis Institutes for BioMedical Research will collaborate with Intellia with R&D activities focusing on using CRISPR ex vivo for engineering chimeric antigen receptor T-cells (CAR-Ts) 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 agreement with Intellia, Novartis receives exclusive rights to develop all collaboration programs focused on engineered CAR-Ts 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, which was an investor in Intellia's $15m series A round a few months ago (alongside Atlas Venture), is increasing its equity investment in Intellia, and is making an upfront payment together with 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. Under the agreement with Caribou, Novartis receives non-exclusive rights to Caribou's CRISPR platform for research conducted during the collaboration and is providing funding for the one-year research program. Novartis is also making an equity investment in the company in the form of a series A financing.
RNAi limitations
Caribou says it is advancing its CRISPR-Cas9 platform in drug target screening and validation to help overcome the limitations of current RNAi-based approaches.
"Traditional RNAi-screening technologies yield only a partial, transient suppression of target gene expression. This process may result in either little or inconsistent changes in a cell or animal model as the siRNA is eliminated from cells over time," explained Caribou in a statement. "By inducing a stable, complete knockout of specific genes, CRISPR-Cas9 may offer a superior means of identifying genes representing attractive therapeutic targets."