Stoke Therapeutics is dedicated to addressing the underlying cause of severe diseases by up-regulating 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. Stoke’s first compound, STK-001, is in clinical testing for the treatment of Dravet syndrome (DS), a severe and progressive genetic epilepsy. DS is one of many diseases caused by haploinsufficiency, in which a loss of ~50% of normal protein levels leads to disease. STK-001 may restore Nav1.1 to physiologic levels and has the potential to be the first disease-modifying therapy to address the genetic cause of DS, thus reducing seizures and non-seizure comorbidities. STK-001 has been granted orphan drug designation by the FDA as a potential new treatment for DS. Stoke’s initial focus is haploinsufficiencies and diseases of the CNS 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: Addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines. Using Stoke’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore protein levels.
Brands:STK-001 is in clinical testing to treat Dravet syndrome and STK-002 is being investigated to treat autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder.