Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines. Using the company’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach Stoke is developing antisense oligonucleotides (ASOs) to selectively restore protein levels.
The company’s first compound, STK-001 is in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Dravet syndrome is one of many diseases caused by a haploinsufficiency, in which a loss of ~50% of normal protein levels leads to disease.
The company is pursuing treatment for a second haploinsufficient disease, autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. Stoke’s initial focus is haploinsufficiencies and diseases of the central nervous system and the eye, although proof of concept has been demonstrated in other organs, tissues, and systems, supporting the company’s belief in the broad potential for its proprietary approach.
Stoke Therapeutics Announces That FDA Will Allow the Administration of a Higher Single Dose of STK-001 (70mg) in th ...– Dosing anticipated to start in the coming weeks – – Company remains on track for its planned data readouts from the Phase 1 ...
Stoke Therapeutics Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Business Updates– Company expects to complete Phase 1/2a studies of STK-001 in children and adolescents with Dravet syndrome in 2023 in order ...
Stoke Therapeutics to Participate at Upcoming March Investor ConferencesBEDFORD, Mass. --(BUSINESS WIRE)--Feb. 27, 2023-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated ...
Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines.