Stoke Therapeutics, Inc. STOK
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Stoke Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing precision genetic medicines for the treatment of severe diseases caused by haploinsufficiency, a condition in which a single functional copy of a gene does not produce enough protein for normal function. The company operates within the biotechnology and biopharmaceutical industries, with a primary emphasis on neurological and genetic disorders. Its core technology platform is based on antisense oligonucleotides designed to selectively increase expression of healthy genes rather than suppress them.
The company’s lead product candidate, zorevunersen, targets mutations in the SCN1A gene and is being developed for Dravet syndrome, a rare and severe genetic epilepsy. Stoke Therapeutics was founded in 2017, emerging from research conducted at Cold Spring Harbor Laboratory, and completed its initial public offering in 2019. Since inception, the company has remained focused on advancing a pipeline of RNA-based medicines addressing unmet medical needs in rare diseases.
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Bedford, MA 01730