Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops novel antisense oligonucleotide (ASO) medicines to treat the underlying causes of severe genetic diseases in the United States.

The company utilizes its proprietary Targeted Augmentation of Nuclear Gene Output to design ASOs to precisely upregulate protein expression.

Its lead clinical candidate is STK-001, which is in phase I/IIa clinical trial to treat Dravet syndrome, a severe and progressive genetic epilepsy; and STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy.

It had entered into a license and collaboration agreement with Acadia Pharmaceuticals Inc. for the discovery, development, and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system.

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.