Rare genetic disease, Autosomal Dominant Optic Atrophy 

Stoke Therapeutics, is a biotechnology company dedicated to addressing the underlying cause of severe diseases by up-regulating 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 is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts.

Drug & Indication	Stage & Event	Catalyst Date
STK-001 Details Dravet syndrome, Epilepsy	Phase 1/2 Data readout	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.
STK-002 (UK) Details Rare genetic disease, Autosomal Dominant Optic Atrophy	Phase 1/2 Initiation	Some catalyst date You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details. You must sign up to see the catalyst details.Sign up now.

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