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.
Stoke Therapeutics Reports First Quarter Financial Results and Provides Business Updates– 2H 2022 clinical data readout on track for patients with Dravet syndrome who received multiple doses of STK-001 (30mg) in P ...
Stoke Therapeutics Presents New In-Vivo Data That Demonstrated Dose-Related Target Engagement and OPA1 Protein Upre ...– Data presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting support the Company’s work ...
Stoke Therapeutics to Present at The Association for Research in Vision and Ophthalmology (ARVO) 2022 Annual Meetin ...– New preclinical data support the ongoing preclinical development of STK-002 for the treatment of autosomal dominant optic a ...
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.