STOK

Stoke Therapeutics, Inc. is a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with ribonucleic acid (RNA)-based medicines. The Company, through its proprietary Targeted Augmentation of Nuclear Gene Output (TANGO) approach, is engaged in developing antisense oligonucleotides (ASOs) to selectively restore protein levels. Its first compound, STK-001, is in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. TANGO aims to restore missing proteins by increasing or stoking protein output from healthy genes, thus compensating for the non-functioning copy of the gene. The Company is pursuing the development of STK-002 for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. The Company’s initial focus is haploinsufficiency and diseases of the central nervous system and the eye.