Stoke Therapeutics

Stoke Therapeutics is a biotechnology company focused on developing RNA-based medicines to address the underlying causes of severe genetic diseases. The company's proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach utilizes antisense oligonucleotides (ASOs) to upregulate protein expression by leveraging the non-mutant copy of genes affected by haploinsufficiency. Stoke's lead candidate, STK-001, is in clinical development for the treatment of Dravet syndrome, a severe genetic epilepsy. STK-001 is designed to increase NaV1.1 protein levels by targeting the SCN1A gene to potentially reduce seizures and improve non-seizure symptoms. In clinical trials, STK-001 has shown promising results, with patients receiving higher doses experiencing substantial reductions in convulsive seizure frequency and improvements in cognition and behavior. The company is also advancing STK-002 for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. Stoke's pipeline includes additional programs targeting other genetic neurodevelopmental diseases. In April 2024, Stoke completed a public offering that raised net proceeds of USD 120.3 million to support its ongoing research and development efforts.

Key customers and partnerships

Stoke Therapeutics has established a collaboration with Acadia Pharmaceuticals to discover, develop, and commercialize novel RNA-based medicines for severe and rare genetic neurodevelopmental diseases of the central nervous system. This partnership, announced in July 2022, includes programs targeting Rett syndrome (MECP2), SYNGAP1 syndrome, and an undisclosed neurodevelopmental target. The collaboration provides Stoke with research support and potential milestone payments. The company is also collaborating with patient advocacy groups and medical experts in the fields of epilepsy and rare genetic disorders to advance its therapeutic programs.