Intellia Therapeutics
Intellia Therapeutics, founded in November 2014, focuses on developing potentially curative genome editing treatments using CRISPR technology, with multiple collaborations and promising clinical trials in progress.
Company History
Intellia Therapeutics was founded in November 2014. Since its inception, the company has focused on developing genome editing treatments using CRISPR technology. Intellia Therapeutics aims to transform the lives of individuals with severe diseases through its potentially curative genome editing solutions.
Mission and Vision
Intellia Therapeutics' mission is to transform the lives of people with severe diseases by developing potentially curative genome editing treatments. The company employs a modular genome editing platform to create diverse in vivo and ex vivo therapeutics, with the goal of addressing unmet medical needs.
Collaborations and Partnerships
Intellia Therapeutics has entered into several strategic collaborations and partnerships to advance its research and development efforts. These include partnerships with Regeneron for CRISPR/Cas9-based therapeutic products, Novartis for ex vivo development, and SparingVision for ocular disease treatments. Additional collaborations involve Kyverna Therapeutics for allogeneic CD19 CAR-T cell therapy, ONK Therapeutics for NK cell therapies, Ospedale San Raffaele for T cell-based cancer therapies, and ReCode Therapeutics for cystic fibrosis gene editing therapies.
Clinical Trials and Pipeline
Intellia Therapeutics has several clinical trials and research initiatives underway. NTLA-2001 is being evaluated in a Phase 1 trial for hereditary ATTR amyloidosis with polyneuropathy and has received FDA clearance to initiate a pivotal Phase 3 trial for ATTR amyloidosis with cardiomyopathy. NTLA-2002 is undergoing studies for hereditary angioedema (HAE) and has clinical data published in the New England Journal of Medicine. Additionally, NTLA-3001 is in the preclinical stage for AATD-associated lung disease, and Intellia is developing CRISPR-based treatments for hemophilia A and B in collaboration with Regeneron.
Technological Innovations
Intellia Therapeutics utilizes a systemic lipid nanoparticle (LNP)-based delivery system for in vivo genome editing. This platform allows for targeted and efficient delivery of CRISPR components to specific tissues. The company's pipeline includes NTLA-2001, which aims to address both hereditary and wild-type ATTR amyloidosis with a single treatment, and NTLA-2002, which employs a knockout edit of the KLKB1 gene to prevent HAE attacks. NTLA-3001 targets the insertion of a functional SERPINA1 gene to address AATD-associated lung disease.