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Find Clinical Drug Pipeline Developments & Deals by Stoke Therapeutics
Stoke intends to use the net proceeds to fund research, clinical and process development and manufacturing of its product candidates, including late-stage development of STK-001, in clinical testing for the treatment of Dravet syndrome and further development of STK-002.
Stoke intends to use the net proceeds to fund research, clinical and process development and manufacturing of its product candidates, including late-stage development of STK-001, in clinical testing for the treatment of Dravet syndrome and further development of STK-002.
STK-001 (zorevunersen sodium) is a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome.
STK-001 is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities related to dravet syndrome.
STK-002 is a proprietary antisense oligonucleotide,designed to restore OPA1 protein expression by upregulating protein production from the non-mutant, wild-type, copy of OPA1 gene, thus slowing or even stopping vision loss in patients with autosomal
dominant optic atrophy.
STK-001 is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities related to dravet syndrome.
STK-001 is an investigational new medicine for the treatment of Dravet syndrome. Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome.
Data presented at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting will support the Company’s work to advance STK-002 as the first potential disease modifying treatment for Autosomal Dominant Optic Atrophy.
STK-002 is a proprietary antisense oligonucleotide, designed to upregulate OPA1 protein expression by leveraging non-mutant copy of OPA1 gene to restore OPA1 protein expression with aim to stop or reverse vision loss in patients with ADOA.
Collaboration of two companies involves to discover, develop and commercialize RNA-based medicines for potential treatment of rare genetic neurodevelopmental diseases, SYNGAP1 syndrome, Rett syndrome, and undisclosed neurodevelopmental target of mutual interest.