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 Presents Data from a Combined Interim Analysis of the Phase 1/2a MONARCH and ADMIRAL Studies of ...– Single and multiple doses of STK-001 up to 45mg were well-tolerated – – 55% median reduction from baseline in convulsive se ...
Stoke Therapeutics to Present Data from the Company’s Dravet Syndrome Program at the American Epilepsy Society 2022 ...– Seven abstracts related to the Company’s work in Dravet syndrome will be presented, including data from a combined interim ...
Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates– Company announces positive interim data from the ongoing Phase 1/2a clinical studies of STK-001 in children and adolescents ...
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.