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Stoke Therapeutics is a clinical-stage biotechnology company pioneering a novel approach to treating severe genetic diseases by precisely upregulating protein expression. Utilizing its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) platform, the company develops antisense oligonucleotides (ASOs) designed to restore target proteins to near-normal levels in diseases caused by haploinsufficiency. Its lead clinical program, zorevunersen (STK-001), is currently in Phase 3 development for the treatment of Dravet syndrome, a severe form of genetic epilepsy. Stoke is also advancing STK-002 for autosomal dominant optic atrophy (ADOA) and exploring additional applications in other rare neurological and ocular conditions.
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