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 the Phase 1/2a MONARCH Study of STK-001 in Children and Adolescents with Drav ...– Single doses of STK-001 up to 30mg and multiple doses of 20mg were well tolerated with no safety concerns related to study ...
Stoke Therapeutics Announces Presentations from the Company’s Dravet Syndrome Program at the American Epilepsy Soci ...– Data from the positive interim analysis of the Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet ...
Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates– Company nominates STK-002 as clinical candidate for the treatment of autosomal dominant optic atrophy (ADOA), the most comm ...
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.