Stoke Therapeutics is betting on a new kind of genetic medicine that aims to treat the root cause of serious childhood seizures and a blinding eye condition, with a lead program edging closer to a first launch and a second program expanding the story beyond one disease. The big question is whether the promising early results hold up in larger trials—and whether safety, insurance coverage, and competition get in the way.Read more
Stoke Therapeutics is chasing treatments for severe genetic diseases, but tougher regulators and watchful insurers could slow the path to market and limit what these drugs can charge. The big question is whether its lead epilepsy program can prove clear, lasting benefits and stay safe over time as competition heats up.Read more
Catalysts About Stoke Therapeutics Stoke Therapeutics is a biotechnology company focused on RNA-based medicines, including zorevunersen for Dravet syndrome. What are the underlying business or industry changes driving this perspective?Read more
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Stoke Therapeutics, Inc. engages in the development of treatments for severe genetic diseases by upregulating protein expression. The company utilizes its proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) approach in developing antisense oligonucleotides (ASOs) to selectively restore protein levels. Its lead product candidates include STK-002, which is in Phase 1 clinical trials for the treatment of autosomal dominant optic atrophy; and Zorevunersen (STK-001), an investigational new medicine for the treatment of Dravet syndrome is being evaluated in phase 3 clinical trials. The company also develops programs focused on various targets, including haploinsufficiency diseases of the central nervous system and eye. It has a license and collaboration with Biogen Inc. for the development and commercialization of zorevunersen medicine for the treatment of Dravet syndrome; and Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of genetic neurodevelopmental diseases. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in May 2016. Stoke Therapeutics, Inc. was incorporated in 2014 and is headquartered in Bedford, Massachusetts.
