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 to Participate in the 2023 Cantor Global Healthcare ConferenceBEDFORD, Mass. --(BUSINESS WIRE)--Sep. 21, 2023-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated ...
Stoke Therapeutics Appoints Ian Smith to its Board of Directors– Mr. Smith brings significant experience in the successful development and commercialization of medicines for rare genet ...
Stoke Therapeutics Presents Data Related to the Ongoing Clinical Development of STK-001 for the Treatment of Dravet ...– Data from multiple ongoing clinical studies of STK-001 show reductions in convulsive seizure frequency and improvements in ...
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.