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[{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stoke Therapeutics's Preclinical Data reveal In-Vitro and In-Vivo Target Engagement and Protein Upregulation in OPA1 Protein Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stoke Therapeutics Announces Publication of Preclinical Data on STK-001 in the Journal Science Translational Medicine","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Neurology","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stoke Therapeutics Announces Plans to Move Forward With Dosing of STK-001 in Children and Adolescents in its Ongoing Phase 1\/2a MONARCH Study for Dravet Syndrome","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Neurology","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stoke Therapeutics To Start Dravet Syndrome Therapy Trial in UK","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Neurology","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stoke Therapeutics Announces Presentations from the Company\u2019s Dravet Syndrome Program at the American Epilepsy Society 2021 Annual Meeting","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Neurology","graph2":"Phase II"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stoke 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Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stoke Therapeutics Presents Data from a Combined Interim Analysis of the Phase 1\/2a MONARCH and ADMIRAL Studies of STK-001 in Children and Adolescents with Dravet Syndrome at the American Epilepsy Society (AES) 2022 Annual Meeting","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Neurology","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Acadia Pharmaceuticals","pharmaFlowCategory":"D","amount":"$967.0 million","upfrontCash":"$60.0 million","newsHeadline":"Acadia Pharmaceuticals and Stoke Therapeutics Announce Collaboration to Pursue Multiple RNA-based Treatments for Severe and Rare Genetic Neurodevelopmental Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stoke Therapeutics Announces That FDA Will Allow the Administration of a Higher Single Dose of STK-001 (70mg) in the Ongoing Phase 1\/2a MONARCH Study of Children and Adolescents with Dravet Syndrome","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Neurology","graph2":"Phase II"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stoke Therapeutics Receives Authorization to Initiate a Phase 1\/2 Study of STK-002 for Autosomal Dominant Optic Atrophy (ADOA) in the United Kingdom","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stoke Therapeutics Announces Positive New Safety & Efficacy Data from Patients Treated with STK-001 in the Phase 1\/2a Studies (MONARCH & ADMIRAL) and the SWALLOWTAIL Open-Label Extension (OLE) Study in Children and Adolescents with Dravet Syndrome","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Neurology","graph2":"Phase II"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","amount":"$75.0 million","upfrontCash":"Undisclosed","newsHeadline":"Stoke Therapeutics Announces Proposed Public Offering","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Neurology","graph2":"Phase II"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","amount":"$125.0 million","upfrontCash":"Undisclosed","newsHeadline":"Stoke Therapeutics Announces Pricing of Upsized $125 Million Public Offering","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Neurology","graph2":"Phase II"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stoke Therapeutics Announces Landmark New Data That Support the Potential for STK-001 to be the First Disease-Modifying Medicine for the Treatment of Patients with Dravet Syndrome","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Neurology","graph2":"Phase II"}]

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            Details:

            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.

            Lead Product(s): Zorevunersen Sodium

            Therapeutic Area: Neurology Product Name: STK-001

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: J.P. Morgan

            Deal Size: $125.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering March 27, 2024

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            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.

            Lead Product(s): Zorevunersen Sodium

            Therapeutic Area: Neurology Product Name: STK-001

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: J.P. Morgan

            Deal Size: $75.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering March 26, 2024

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            Details:

            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.

            Lead Product(s): Zorevunersen Sodium

            Therapeutic Area: Neurology Product Name: STK-001

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 25, 2024

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            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.

            Lead Product(s): STK-001

            Therapeutic Area: Neurology Product Name: STK-001

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 25, 2023

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            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.

            Lead Product(s): STK-002

            Therapeutic Area: Genetic Disease Product Name: STK-002

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 25, 2023

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            Details:

            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.

            Lead Product(s): STK-001

            Therapeutic Area: Neurology Product Name: STK-001

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 15, 2023

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            Details:

            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.

            Lead Product(s): STK-001

            Therapeutic Area: Neurology Product Name: STK-001

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 02, 2022

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            Details:

            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.

            Lead Product(s): STK-002

            Therapeutic Area: Genetic Disease Product Name: STK-002

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 02, 2022

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            Details:

            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.

            Lead Product(s): STK-002

            Therapeutic Area: Genetic Disease Product Name: STK-002

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 07, 2022

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            Details:

            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.

            Lead Product(s): RNA-based Therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Preclinical Product Type: Large molecule

            Partner/Sponsor/Collaborator: Acadia Pharmaceuticals

            Deal Size: $967.0 million Upfront Cash: $60.0 million

            Deal Type: Collaboration January 10, 2022

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