Stoke Therapeutics

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Stoke Therapeutics is developing a new category of RNA medicines focused on upregulating protein expression from functional genes to treat severe genetic diseases. The company's lead program, zorevunersen (STK-001), is in Phase 3 for Dravet syndrome, with recent data published in The New England Journal of Medicine demonstrating potential disease modification. Stoke's strategy leverages its proprietary TANGO platform to address haploinsufficiency disorders, a large class of genetic conditions, and expand into other areas of high unmet medical need. Its $1.91B valuation reflects significant investor confidence in this novel therapeutic approach.

Proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform uses antisense oligonucleotides (ASOs) to modulate RNA splicing and increase production of functional proteins from healthy genes, targeting the root cause of haploinsufficiency and other genetic disorders.