Intellia Therapeutics

Intellia Therapeutics, established in November 2014, is dedicated to developing potentially curative genome editing treatments using CRISPR technology. The company employs a modular genome editing platform to create diverse in vivo and ex vivo therapeutics, aiming to transform the lives of people with severe diseases. Intellia’s mission is to develop treatments that can potentially cure these conditions. The company has formed several strategic collaborations, including partnerships with Regeneron for CRISPR/Cas9-based therapeutic products, Novartis for ex vivo development, and SparingVision for ocular diseases. Additionally, Intellia has launched AvenCell in collaboration with Blackstone Life Sciences, Cellex Cell Professionals GmbH, and GEMoaB, and has agreements with Kyverna Therapeutics for CD19 CAR-T cell therapy and ONK Therapeutics for NK cell therapies for cancer treatment. Intellia is also working with Ospedale San Raffaele to engineer T cell-based cancer therapies and with ReCode Therapeutics to develop gene editing therapies for cystic fibrosis. The company’s pipeline includes NTLA-2001, which is in a Phase 1 clinical trial for hereditary ATTR amyloidosis with polyneuropathy and has received FDA clearance for a pivotal Phase 3 trial for ATTR amyloidosis with cardiomyopathy. NTLA-2002 is being studied for hereditary angioedema (HAE) and has shown positive interim results, with clinical data published in the New England Journal of Medicine in January 2024. NTLA-3001 is in the research and preclinical stage for AATD-associated lung disease. Intellia is also developing CRISPR-based treatments for hemophilia A and B in collaboration with Regeneron. The company utilizes a systemic lipid nanoparticle (LNP)-based delivery system for in vivo genome editing and focuses on engineering T cell therapies for oncological and immunological diseases. NTLA-2001 aims to address both hereditary and wild-type ATTR amyloidosis with a single course of treatment, while NTLA-2002 employs a knockout edit of the KLKB1 gene to reduce plasma kallikrein activity and prevent HAE attacks. NTLA-3001 targets the insertion of a functional SERPINA1 gene to address AATD-associated lung disease.