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Stoke Therapeutics, Inc. is a biotechnology company focused on addressing the underlying causes of severe genetic diseases by upregulating protein expression with ribonucleic acid-based medicines. Using its Targeted Augmentation of Nuclear Gene Output (TANGO) approach, the Company is 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. STK-001 is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both the occurrence of seizures and non-seizure comorbidities. Its STK-002 is a ASO in preclinical development for the treatment of Autosomal Dominant Optic Atrophy (ADOA). STK-002 is designed to upregulate OPA1 protein expression by leveraging the non-mutant (wild-type) copy of the OPA1 gene.
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