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Intellia Therapeutics is a gene editing company focused on the development of Crispr/Cas9-based therapeutics. Crispr/Cas9 stands for Clustered Regularly Interspaced Short Palindromic Repeats (Crispr)/Crispr-associated protein 9 (Cas9), which is a revolutionary technology for precisely altering specific sequences of genomic DNA. Intellia is focused on using this technology to treat genetically defined diseases. It's evaluating multiple gene editing approaches using in vivo and ex vivo therapies to address diseases with high unmet medical needs, including ATTR amyloidosis, hereditary angioedema, sickle cell disease, and immuno-oncology. Intellia has formed collaborations with several companies to advance its pipeline, including narrow-moat Regeneron and wide-moat Novartis.
OPKO Health Inc is a diversified biotechnology company that operates pharmaceutical and diagnostic development programs. Its segments include pharmaceutical and diagnostics. The Pharmaceutical segment consists of its pharmaceutical operations in Chile, Mexico, Ireland, Israel, Spain, Ecuador, France, the United States, and its pharmaceutical research and development operations. The Diagnostics segment consists of clinical and genomics laboratory operations through BioReference and point-of-care operations. It generates the majority of its revenue from the Diagnostics segment.