Profile: Intellia Therapeutics Inc (NTLA.A)
Intellia Therapeutics, Inc., incorporated on May 7, 2014, is a genome editing company. The Company is focused on developing therapeutics utilizing a biological tool known as CRISPR/Cas9. The CRISPR/Cas9 genome editing system includes two components: the Cas9 protein and the guide RNA sequence. The Cas9 protein acts like a pair of molecular scissors that initiates the natural cellular repair process to knockout, repair or insert a gene. The guide RNA sequence recognizes and directs the Cas9 to a specific target deoxyribonucleic acid (DNA) sequence.
The Company's sentinel in vivo programs focus on the use of Lipid Nanoparticle (LNPs) for delivery of the CRISPR/Cas9 complex to the liver. The Company's in vivo pipeline includes programs targeting transthyretin amyloidosis (ATTR), alpha-1 antitrypsin deficiency (AATD), hepatitis B virus (HBV) and inborn errors of metabolism (IEMs). The Company's sentinel ex vivo programs include programs focused on chimeric antigen receptor T cells (CAR T cells) and hematopoietic stem cells (HSCs), the stem cells from which all of the various types of blood cells originate. The Company's division, eXtellia Therapeutics, is focused on the application of CRISPR/Cas9 genome editing in the fields of immuno-oncology and autoimmune and inflammatory diseases. The Company's subsidiary is Intellia Securities Corp. As of September 30, 2016, the Company had not generated any revenue.
The Company competes with CRISPR Therapeutics, Inc., Casebia Therapeutics, Editas Medicine, Inc., Tracr Hematology Limited, bluebird bio, Inc., Cellectis S.A., Poseida, Inc., Precision BioSciences, Inc., Sangamo BioSciences and Juno Therapeutics, Inc.
Intellia Therapeutics Inc
40 Erie St Ste 130
CAMBRIDGE MA 02139-4254