Stoke Therapeutics Inc. (STOK)

Profile

https://www.stoketherapeutics.com

Stoke Therapeutics Inc. is a biotechnology company focused on developing RNA-based medicines to treat the underlying causes of severe genetic diseases. It employs its proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) platform, which uses antisense oligonucleotides to upregulate protein expression from healthy genes, compensating for mutated copies in conditions known as autosomal dominant haploinsufficiencies. This approach targets tissues including the central nervous system, eye, kidney, and liver. The company's lead investigational medicine, zorevunersen (STK-001), addresses Dravet syndrome, a severe genetic epilepsy characterized by frequent seizures, intellectual disability, and developmental delays, with ongoing Phase 3 clinical studies in collaboration with Biogen. Additional efforts explore TANGO ASOs for autosomal dominant optic atrophy and other severe diseases. Stoke Therapeutics Inc. maintains a pipeline aimed at pioneering disease-modifying therapies. Founded in 2014 and headquartered in Bedford, Massachusetts, the company drives innovation in precision medicine for genetic disorders.