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Stoke Therapeutics Inc is a biotechnology company dedicated to restoring protein expression by harnessing the body's potential with RNA medicine. Its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to restore naturally-occurring protein levels selectively. Its first medicine in development, zorevunersen, has demonstrated the potential for disease modification in patients with Dravet syndrome. The company provides early programs focused on multiple targets, including haploinsufficiency diseases of the central nervous system and eye, to up-regulate the protein expression of the underlying disease gene in a mutation-agnostic manner.
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.