Stoke Therapeutics, Inc. are pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression. They are 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. They designed their lead product candidate, STK-001, to treat Dravet syndrome, a severe and progressive genetic epilepsy. With a well-defined patient population based on routine genetic testing and learnings from recently approved drugs for the treatment of Dravet syndrome to inform the clinical and regulatory pathways for STK-001, they anticipate an efficient clinical program for STK-001. They plan to submit an investigational new drug application, or IND, for STK-001 by early 2020 and expect to initiate a Phase 1/2 clinical trial in the first half of 2020. They intend to nominate a second candidate to treat an additional genetic disease for preclinical development by the first half of 2020. Stoke Therapeutics, Inc. were incorporated under the laws of the State of Delaware in June 2014 under the name ASOthera Pharmaceuticals, Inc. They subsequently changed their name to Stoke Therapeutics, Inc. on May 18, 2016. Their principal executive office is
located at 45 Wiggins Avenue, Bedford, Massachusetts, 01730.