Intellia Therapeutics Inc. is an American clinical-stage biotechnology company with a focus on gene editing and the development of therapeutics based on a unique biological gene editing tool called CRISPR, a technology for genome editing, which is the process of altering selected sequences of genomic deoxyribonucleic acid (DNA). The company is engaged in developing products aimed at helping patients suffering from various medical conditions, such as cancer, genetic disorders, viral infections and inflammatory disorders. The company was founded in 2014 and is headquartered in Cambridge, Massachusetts.
The firm is engaged in both in vivo programs, which use intravenously administered CRISPR as therapy, enabling precise editing of disease-causing genes directly within specific target tissues, and ex vivo programs, which use CRISPR to create new therapies using engineered human cells, which can then be administered to treat cancer and autoimmune diseases.
Each treatment consists of a particle specifically designed to deliver to the liver a simple, two-part genome editing system: the messenger RNA that encodes the Cas9 protein and guides RNA to the target gene. The firm’s stock is traded on the NASDAQ where it is listed under the stock ticker NTLA.
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