CSBio CSBio

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Hyperinsulinism","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Rezolute"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CHMP Issues Negative Opinion for Renewal of Conditional Marketing Authorization for Translarna\u2122 (ataluren) Following Re-examination Procedure","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"PTC Therapeutics"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not 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molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Synlogic"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Jefferies","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"KalVista Pharmaceuticals Announces Proposed Public Offering of Common Stock and Pre-Funded Warrants","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"KalVista Pharmaceuticals"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Announces Positive Results From Pivotal Trials of Vanzacaftor\/Tezacaftor\/Deutivacaftor, Next-in-Class Triple Combination Treatment for Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"Vertex Pharmaceuticals"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"KalVista Pharmaceuticals Reports Phase 3 KONFIDENT Trial Meets All Endpoints for Sebetralstat as First Oral On-demand Therapy for Hereditary Angioedema","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Small molecule","graph2":"KalVista Pharmaceuticals"},{"orgOrder":0,"company":"Mabxience","sponsor":"Biosidus 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Pharmaceuticals"},{"orgOrder":0,"company":"Capricor Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Capricor Therapeutics Announces Upcoming Type-B Meeting with the FDA to Discuss Commercial Manufacturing Planning with an Aim to Expedite BLA Pathway for CAP-1002 in Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Capricor Therapeutics"}]

Find Drugs for Genetic Disease in Phase III Clinical Development in UNITED STATES

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            SAR-439774 (fitusiran) is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.

            Lead Product(s): Givosiran

            Therapeutic Area: Genetic Disease Product Name: SAR-439774

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 04, 2023

            Sanofi Company Banner

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            BIVV001 (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy with the potential to deliver near-normal factor activity levels for most of the week, extending bleed protection in a once-weekly dose for people with haemophilia A.

            Lead Product(s): Efanesoctocog Alfa

            Therapeutic Area: Genetic Disease Product Name: BIVV001

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 25, 2023

            Sanofi Company Banner

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            BIVV001 (efanesoctocog alfa) is a novel and investigational recombinant factor VIII therapy that is designed to extend protection from bleeds with once-weekly prophylactic dosing for people with hemophilia A.

            Lead Product(s): Efanesoctocog Alfa

            Therapeutic Area: Genetic Disease Product Name: BIVV001

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 30, 2022

            Sanofi Company Banner

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            Investigational once-weekly BIVV001 (efanesoctocog alfa) prophylaxis met the primary efficacy endpoint providing clinically meaningful bleed protection for people with severe hemophilia A.

            Lead Product(s): Efanesoctocog Alfa

            Therapeutic Area: Genetic Disease Product Name: BIVV001

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Swedish Orphan Biovitrum AB

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 10, 2022

            Sanofi Company Banner

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            Details:

            SAR-439774 (fitusiran) is an investigational, subcutaneously administered small interference RNA therapeutic in development for the prophylactic treatment of people with hemophilia A or B, with or without inhibitors.

            Lead Product(s): Givosiran

            Therapeutic Area: Genetic Disease Product Name: SAR-439774

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 10, 2022

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            Details:

            BIVV001 (efanesoctocog alfa) is a novel and investigational factor VIII therapy designed to provide normal to near-normal factor activity levels for the majority of the week in a once-weekly prophylactic treatment regimen.

            Lead Product(s): Efanesoctocog Alfa

            Therapeutic Area: Genetic Disease Product Name: BIVV001

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Swedish Orphan Biovitrum AB

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 01, 2022

            Sanofi Company Banner

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            Details:

            The study met the primary endpoint, showing a clinically meaningful prevention of bleeds in people with severe hemophilia A receiving weekly prophylaxis with BIVV001 (efanesoctocog alfa) over a period of 52 weeks.

            Lead Product(s): Efanesoctocog Alfa

            Therapeutic Area: Genetic Disease Product Name: BIVV001

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Swedish Orphan Biovitrum AB

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 09, 2022

            Sanofi Company Banner

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            Details:

            Data from phase 3 studies achieved primary and secondary endpoints for ALN-AT3SC (Fitusiran) prophylaxis demonstrated significant and clinically meaningful improvements in bleed protection across all study populations.

            Lead Product(s): Givosiran

            Therapeutic Area: Genetic Disease Product Name: ALN-AT3SC

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 14, 2021

            Sanofi Company Banner

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            Details:

            Efanesoctocog alfa, previously known as BIVV001, is an investigational factor VIII replacement therapy that has the potential to transform therapy and provide high sustained factor activity levels for people with hemophilia A.

            Lead Product(s): Efanesoctocog alfa

            Therapeutic Area: Genetic Disease Product Name: BIVV001

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Sanofi

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 18, 2021

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            Details:

            Sanofi will resume fitusiran dosing in ongoing U.S. adolescent and adult clinical studies. Fitusiran is an investigational, small interference RNA therapy in development for the treatment of people with hemophilia A or B, with or without inhibitors.

            Lead Product(s): Givosiran

            Therapeutic Area: Genetic Disease Product Name: ALN-AT3SC

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 10, 2020

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