STOK

Stoke Therapeutics, Inc., an early-stage biopharmaceutical company, develops novel antisense oligonucleotide (ASO) medicines to treat the underlying causes of severe genetic diseases in the United States. The company utilizes its proprietary Targeted Augmentation of Nuclear Gene Output to design ASOs to precisely upregulate protein expression. Its lead clinical candidate is STK-001, which is in phase I/IIa clinical trial to treat Dravet syndrome, a severe and progressive genetic epilepsy; and STK-002, which is in preclinical stage for the treatment of autosomal dominant optic atrophy.