CSBio CSBio

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[{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Priority Review for Avalglucosidase Alfa, a Potential New Therapy for Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Pharnext","sponsor":"Not Applicable ","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharnext Receives Promising Innovative Medicine Designation for PXT3003 ","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharnext"},{"orgOrder":0,"company":"GenSight Biologics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GenSight Biologics Announces Presentation of Bilateral Visual Recovery From GS010 Phase III Trials","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"GenSight Biologics"},{"orgOrder":0,"company":"Ipsen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ipsen Demonstrates Continued Commitment to Rare Diseases with Eight Abstracts Accepted at ENDO 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular 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molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sanofi Announces Long-Term Efficacy and Safety Data for Fitusiran from Interim Analysis of Phase 2 Extension Study in Hemophilia A and B","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Data presented at ISTH Virtual Congress highlights Sanofi\u2019s commitment to advancing treatments for people with rare blood 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to Present Results from Move, the First Global Phase III Trial in Fibrodysplasia Ossificans Progressiva (fop), at Asbmr 2020","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Ipsen"},{"orgOrder":0,"company":"Sanofi","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NEJM Publishes Positive Final Results from Phase 1\/2a Study of BIVV001 in People with Severe Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Ipsen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ipsen To Present New Insights at ASBMR For Potential Treatment of Ultra-Rare Disease Fibrodysplasia Ossificans Progressiva, Including Global Phase III MOVE Trial Results","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Ipsen"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"EMA Accepts Sanofi's Regulatory Submission for Avalglucosidase Alfa for Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"EMA Accepts Regulatory Submission for Avalglucosidase Alfa, a Potentially new Standard of Care Enzyme Replacement Therapy for Pompe disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Inventiva Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inventiva Receives FDA Fast Track Designation in MPS VI for its Clinical-Stage Asset Odiparci","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Inventiva Pharma"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Global Dosing Hold in Fitusiran Trials Initiated by Sanofi Genzyme to Investigate New Adverse Events","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sanofi to Resume Dosing in Fitusiran Clinical Studies in the U.S","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large 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Charcot-Marie-Tooth Disease Type 1A After 5 Years of Total Trial Time","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"May 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharnext"},{"orgOrder":0,"company":"Sanofi","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Efanesoctocog Alfa Breakthrough Therapy Designation for Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Sanofi","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pivotal Data 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Charcot-Marie-Tooth Disease Type 1A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharnext"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sanofi to present new clinical data reinforcing novel therapies across rare blood disorders at ASH 2022","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NEJM Publishes Once-Weekly Efanesoctocog Alfa Phase 3 Data Demonstrating Its Potential to Transform the Treatment Landscape for People with Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Sanofi"},{"orgOrder":0,"company":"GenSight Biologics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GenSight Biologics Confirms Sustained Efficacy and Safety of Bilateral LUMEVOQ\u00ae Injections at 3-Year Follow-Up of REFLECT Phase III Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"GenSight Biologics"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not 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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 April 04, 2023

            Sanofi Company Banner

            TMF Summit 2024

            Not Confirmed

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            Details:

            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

            TMF Summit 2024

            Not Confirmed

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            Details:

            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

            TMF Summit 2024

            Not Confirmed

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            Details:

            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

            TMF Summit 2024

            Not Confirmed

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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

            Sanofi Company Banner

            TMF Summit 2024

            Not Confirmed

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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

            TMF Summit 2024

            Not Confirmed

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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

            TMF Summit 2024

            Not Confirmed

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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

            TMF Summit 2024

            Not Confirmed

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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

            Sanofi Company Banner

            TMF Summit 2024

            Not Confirmed

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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

            Sanofi Company Banner

            TMF Summit 2024

            Not Confirmed

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