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Stoke Therapeutics is a clinical-stage biotechnology company dedicated to addressing the underlying causes of severe genetic diseases by upregulating gene expression. Utilizing its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform, the company develops antisense oligonucleotides (ASOs) designed to increase the production of functional proteins in cases of haploinsufficiency. Its lead clinical candidate, STK-001, is currently in Phase 1/2 clinical trials for the treatment of Dravet syndrome, a severe and progressive form of epilepsy. The company is also advancing STK-002 for the treatment of autosomal dominant optic atrophy (ADOA), a leading cause of inherited vision loss. Stoke's unique approach focuses on restoring protein levels to normal physiological ranges, offering a potential disease-modifying treatment for various haploinsufficient conditions.
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