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Stoke Therapeutics develops RNA medicines intended to restore protein expression, using its TANGO approach and antisense oligonucleotides for diseases caused by insufficient gene output. Its lead investigational medicine, zorevunersen, is in development for Dravet syndrome, with earlier work directed at rare genetic neurodevelopmental and other haploinsufficiency diseases.
Company news commonly covers zorevunersen clinical data, EMPEROR study updates, financial results, capital resources, collaboration activity, board and governance changes, equity inducement grants under Nasdaq rules, and healthcare conference presentations.
Stoke Therapeutics (Nasdaq: STOK) is advancing its Phase 1/2a MONARCH study for STK-001, targeting Dravet syndrome, after receiving FDA approval to add higher dose levels for evaluation. The study will now assess doses of 10 mg, 20 mg, and 30 mg, with studies above 30 mg on hold. No adverse effects were noted in preclinical repeat-dose tests. The company aims to submit a protocol amendment for a multiple ascending dose segment. STK-001, an investigational drug, aims to adjust NaV1.1 protein levels, potentially modifying the disease's course.