Stoke’s proprietary proprietary research platform, TANGO, has broad therapeutic potential. Stoke’s initial focus are diseases of the central nervous system and the eye that are caused by a loss of ~50% of normal protein levels (haploinsufficiency).
Our lead clinical program is focused on Dravet syndrome, a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures that usually begin within the first year of life. A clinical program is also underway for Autosomal Dominant Optic Atrophy (ADOA) a severe, progressive optic nerve disorder.
Dravet Syndrome
Dravet syndrome occurs globally and is not concentrated in a particular geographic area or ethnic group. Currently, it is estimated that up to 38,000 people are living with Dravet syndrome in the U.S., UK, EU-4 and Japan.
Autosomal Dominant Optic Atrophy (ADOA)
ADOA is the most common inherited optic nerve disorder seen in clinical practice.
SYNGAP1
SYNGAP1 is a rare neurological disorder characterized by moderate to severe intellectual disability that is evident in early childhood.
Our Pipeline
Using the Company’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore protein levels.