Intellia and ReCode partner to develop gene-editing therapies against CF

The collaboration includes Intellia’s CRISPR-based gene editing platform and ReCode’s selective organ targeting (SORT) lipid nanoparticle (LNP) delivery platform. It aims to prevent one or more CF disease-causing gene mutations.  

As per the agreement, Intellia will be in control of creating research-grade parts for the experimental treatments as well as the editing strategy, whereas ReCode will lead preclinical and clinical development. Additionally, global commercialization will be led by ReCode, while Intellia will be responsible for commercialization for certain programs in the US. 

“One of Intellia’s strategic priorities through 2026 is extending the reach of CRISPR-based gene editing to tissues outside the liver,” a spokesperson for Intellia told BioProcess Insider. 

“Our new collaboration with ReCode is focused on accelerating development of CRISPR-based treatments targeting genes in the lung for cystic fibrosis. Building on our success with CRISPR/Cas9, we have made important progress with our proprietary DNA writing technology. We are excited to combine our gene editing expertise and platform with ReCode’s novel lung directed LNP delivery platform.” 

According to the spokesperson, the companies will focus initial research efforts on therapeutic approaches that address CF for patients who have limited or no treatment options available, with the opportunity to expand the scope of the collaboration in later phases. 

Moreover, there is no upfront payment, and Intellia will be eligible to receive pre-specified development and commercial milestone payments, as well as royalties on potential sales. 

Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause CF, a hereditary illness that causes thick mucus to build up in the lungs, digestive tract, and other organs. This has severe consequences including respiratory failure, potentially fatal infections. CFTR protein helps to maintain the balance of salt and water on many surfaces in the body, such as the surface of the lung. 

Intellia had previously combined its technology with Blackstone and Cellex to launch a CAR-T biotech to develop chimeric antigen receptor (CAR) T-cell therapies. The agreement, 

signed in 2021, combined Intellia’s allogeneic cell platform and CRISPR cell engineering with Cellex subsidiary GEMoaB’s switchable universal CAR-T platforms – UniCAR and RevCAR – to develop a range of therapies against various cancers, including solid tumors.