Stoke Therapeutics is pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression. The company is developing novel antisense oligonucleotide, or ASO, medicines that target ribonucleic acid, or RNA, and modulate precursor-messenger RNA, or pre-mRNA, splicing to upregulate protein expression where needed and with appropriate specificity to near normal levels. They utilize their proprietary technology platform, Targeted Augmentation of Nuclear Gene Output, or TANGO, to design ASOs to upregulate the expression of protein by individual genes in a patient. Their approach is designed to allow them to deliver in a highly precise, durable and controlled manner disease-modifying therapies to a broad range of relevant tissues, including the central nervous system, or CNS, eye, kidney and liver. The company designed their lead product candidate, STK-001, to treat Dravet syndrome, a severe and progressive genetic epilepsy.