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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 Appoints Eric Olson, MS, MBA as Chief Business Officer

September 4, 2024

– Mr. Olson brings two decades of successful corporate business development experience with an emphasis on rare diseases and ...

Stoke Therapeutics to Present at Upcoming Investor Conferences in September

September 3, 2024

BEDFORD, Mass. --(BUSINESS WIRE)--Sep. 3, 2024-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated t ...

Stoke Therapeutics to Present Encore Data at the 15th European Epilepsy Congress that Demonstrate the Potential for ...

September 3, 2024

– First presentation of landmark data from Phase 1/2a and OLE studies of zorevunersen that showed substantial reductions in s ...

Corporate 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.

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