Stoke’s proprietary proprietary research platform, TANGO, has broad therapeutic potential. Our ongoing research programs currently focus on two severe diseases.
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. We are also examining a preclinical target 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.
Rett Syndrome
Rett syndrome is a rare, debilitating neurological disorder that occurs primarily in females following apparently normal development for the first six months of life.
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.