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Arcturus Therapeutics Holdings Inc is an RNA medicines company focused on opportunities in rare diseases with a current focus on liver and respiratory diseases. It operates in the research and development segment of medical applications for nucleic acid-focused technology. The company's product pipeline includes LUNAR-OTC, LUNAR-CF, LUNAR-FLU and LUNAR-COV19.
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.