Description

Intellia Therapeutics, Inc. is a genome editing company dedicated to discovering and developing therapeutic solutions. Its in vivo pipeline features leading candidates such as NTLA-2001, currently undergoing Phase 1 clinical evaluation for transthyretin amyloidosis, and NTLA-2002, designed to address hereditary angioedema. The company also pursues a range of liver-targeted initiatives, addressing conditions including hemophilia A and B, hyperoxaluria Type 1, and alpha-1 antitrypsin deficiency. Complementing their in vivo efforts, Intellia's ex vivo pipeline includes NTLA-5001 for acute myeloid leukemia. Additionally, they have proprietary programs focused on engineering cell-based therapies for a spectrum of oncological and autoimmune diseases. Central to their platform is the versatile Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. Intellia maintains a network of strategic collaborations, including agreements with Novartis Institutes for BioMedical Research to engineer hematopoietic stem cells for sickle cell disease treatment, and with Regeneron Pharmaceuticals for co-developing potential therapies for hemophilia A and B. Further partnerships involve Ospedale San Raffaele, and a strategic alliance with SparingVision SAS to create novel genomic medicines using CRISPR/Cas9 for ocular diseases. Founded in 2014 as AZRN, Inc., the company is headquartered in Cambridge, Massachusetts.