Corporate Profile
Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines. Using the company’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach Stoke is developing antisense oligonucleotides (ASOs) to selectively restore protein levels.
The company’s first compound, zorevunersen (STK-001) is in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Dravet syndrome is one of many diseases caused by a haploinsufficiency, in which a loss of ~50% of normal protein levels leads to disease.
The company is pursuing treatment for a second haploinsufficient disease, autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. Stoke’s initial focus is haploinsufficiencies and diseases of the central nervous system and the eye, although proof of concept has been demonstrated in other organs, tissues, and systems, supporting the company’s belief in the broad potential for its proprietary approach.
Press Releases
Stoke Therapeutics Announces Alignment with Global Regulatory Agencies and Plans to Initiate a Phase 3 Study of Zor ...
January 7, 2025
– Alignment achieved with U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA) and Japan’s Pharmaceutical ...Stoke Therapeutics to Host Webcast to Discuss Successful Global Regulatory Alignment for a Phase 3 Study of Zorevun ...
January 6, 2025
BEDFORD, Mass. --(BUSINESS WIRE)--Jan. 6, 2025-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated t ...Stoke Therapeutics Presents New Open-Label Extension (OLE) Study Data That Further Support the Potential for Zorevu ...
December 6, 2024
– Substantial and durable reductions in convulsive seizure frequency observed in patients treated with 2 or 3 doses of 70mg f ...Featured Presentation
Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines.
