Corporate Profile
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.
Press Releases
Stoke Therapeutics Presents Data From Multiple Studies of Children and Adolescents With Dravet Syndrome at the Amer ...
December 1, 2023
– Data from clinical studies of STK-001 demonstrated clinical benefit for patients ages 2 to 18 years old, including reductio ...Stoke Therapeutics to Present Data from the Company’s Dravet Syndrome Clinical Program at the American Epilepsy Soc ...
November 21, 2023
– New data include 24-month results from the largest and longest prospective natural history study of children and adolescent ...Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates
November 7, 2023
– STK-001: Q1 2024 readout expected to include end of Phase 1/2a study results and longer-term data on the effects of repeat ...Corporate Presentation
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.
