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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 Enrolls First Patient in a Natural History Study of People Living with Autosomal Dominant Optic ...

August 25, 2022

– ADOA is primarily caused by mutations in the OPA1 gene that result in progressive and irreversible vision loss in both eyes ...

Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates

August 8, 2022

– In the fourth quarter, the Company plans to announce interim safety, pharmacokinetic (PK), and cerebrospinal fluid (CSF) dr ...

Stoke Therapeutics to Present at the 2022 Jefferies Healthcare Conference

June 1, 2022

BEDFORD, Mass. --(BUSINESS WIRE)--Jun. 1, 2022-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated t ...

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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