Intellia Therapeutics, Inc. is a biotechnology firm dedicated to advancing therapeutic treatments through its expertise in genome editing. The company's pipeline includes several in vivo (administered within the body) programs. NTLA-2001 is currently undergoing a Phase 1 clinical trial for transthyretin amyloidosis, while NTLA-2002 targets hereditary angioedema. Additionally, Intellia is developing various other liver-focused therapies for conditions such as hemophilia A and B, hyperoxaluria Type 1, and alpha-1 antitrypsin deficiency. Its ex vivo (processed outside the body) pipeline features NTLA-5001, a candidate for acute myeloid leukemia. The company is also progressing proprietary programs focused on creating engineered cell therapies to address diverse oncological and autoimmune disorders. At the core of Intellia's technology is the Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. Intellia Therapeutics, Inc. has established multiple licensing and collaboration agreements. These include a partnership with Novartis Institutes for BioMedical Research, Inc. to engineer hematopoietic stem cells for treating sickle cell disease, and a co-development agreement with Regeneron Pharmaceuticals, Inc. for potential products against hemophilia A and B. The company also collaborates with Ospedale San Raffaele and holds a strategic alliance with SparingVision SAS to develop novel genomic medicines for ocular diseases, leveraging CRISPR/Cas9 technology. The company, initially known as AZRN, Inc., was founded in 2014 and operates from its headquarters in Cambridge, Massachusetts.
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