Stoke Therapeutics, Inc. — Investor Relations & Filings

Stoke Therapeutics, Inc. is a biotechnology company focused on developing treatments for severe genetic diseases by addressing their underlying cause. The company utilizes its proprietary research platform, TANGO (Targeted Augmentation of Nuclear Gene Output), an RNA-based approach designed to restore protein levels. This technology works by upregulating protein expression from the healthy copy of a gene to compensate for a mutated or non-functioning copy, a mechanism particularly suited for haploinsufficient diseases. Stoke's lead investigational medicine, zorevunersen (STK-001), is in late-stage clinical development for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. The company is also advancing its platform to treat other genetic conditions, including autosomal dominant optic atrophy (ADOA).