Stoke Therapeutics, Inc.

Stoke Therapeutics, Inc. is a biopharmaceutical company dedicated to developing novel treatments for severe genetic diseases by addressing the root cause of protein deficiency. The company's core innovation lies in its proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) platform, which leverages antisense oligonucleotides (ASOs) to selectively increase the expression of target proteins. This approach aims to restore normal protein levels, offering a potentially transformative therapeutic strategy for a range of debilitating conditions. Stoke's pipeline includes promising candidates such as STK-002, currently in preclinical development for autosomal dominant optic atrophy, and zorevunersen (STK-001), an investigational medicine for Dravet syndrome that is advancing through Phase I/II clinical trials. Beyond these lead programs, the company is actively exploring TANGO's application in other haploinsufficiency diseases affecting the central nervous system and the eye. Strategic collaborations are a key component of Stoke's business model, with significant partnerships in place with Biogen Inc. for the development and commercialization of zorevunersen and with Acadia Pharmaceuticals Inc. for the discovery and development of new RNA-based medicines targeting genetic neurodevelopmental disorders. This dual approach of internal pipeline advancement and strategic alliances positions Stoke to address a significant unmet medical need in rare genetic diseases.