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BioCryst Pharmaceuticals Inc is a biotechnology company involved mainly in the research and development of novel small-molecule drugs, to block key enzymes involved in infectious and inflammatory diseases. Biocryst's research is based on multiple scientific disciplines like biology, computer modeling, and medicinal chemistry. Its products and candidates target the following therapeutic areas: acute uncomplicated influenza; uncomplicated seasonal influenza; hereditary angioedema; filoviruses, including the Ebola and Marburg viruses; and oncology.
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.