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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, 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 Reports First Quarter Financial Results and Provides Business Updates

May 4, 2023

– Company on-track to report new data from the ongoing Phase 1/2a clinical studies of STK-001 in children and adolescents wit ...

Stoke Therapeutics to Present at the BofA Securities Health Care Conference

May 3, 2023

BEDFORD, Mass. --(BUSINESS WIRE)--May 3, 2023-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to ...

Stoke Therapeutics Receives Authorization to Initiate a Phase 1/2 Study of STK-002 for Autosomal Dominant Optic Atr ...

April 25, 2023

– Company advancing second TANGO ASO into the clinic – – STK-002 has the potential to be the first disease-modifying therapy ...

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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