Intellia Therapeutics Azione

Intellia Therapeutics, Inc. is a clinical-stage genome editing company, which is focused on developing curative therapeutics using Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) technology. CRISPR/Cas9 is a technology for genome editing, the process of altering selected sequences of genomic deoxyribonucleic acid. It is focused on leveraging its modular platform to advance in vivo and ex vivo therapies for diseases with high unmet need. Its lead in vivo candidate, NTLA-2001, is for the treatment of transthyretin (ATTR) amyloidosis, as well as NTLA-2002 for the treatment of hereditary angioedema (HAE) are the first CRISPR/Cas9-based therapy candidates to be administered systemically, via intravenous (IV) infusion, for precision editing of a gene in a target tissue in humans. It is also developing ex vivo applications to address immuno-oncology and autoimmune diseases.