Disease Areas

Our compounds are designed to address the underlying cause of severe diseases.

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 affects an estimated 35,000 people in the United States, Canada, Japan, Germany, France and the UK.

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 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.