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Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"BridgeBio Pharma \/ Bayer AG","highestDevelopmentStatusID":"10","companyTruncated":"BridgeBio Pharma \/ Bayer AG"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Ionis Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Eplontersen Sodium","moa":"TTR mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"AstraZeneca","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"AstraZeneca \/ Ionis Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"AstraZeneca \/ Ionis Pharmaceuticals"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Eplontersen","moa":"ATTR protein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Ionis Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Ionis Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Ionis Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Onasemnogene Abeparvovec","moa":"SMN1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Novartis Pharmaceuticals Corporation","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Novartis Pharmaceuticals Corporation \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Novartis Pharmaceuticals Corporation \/ Inapplicable"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Apitegromab","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Scholar Rock \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Inapplicable"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Apitegromab","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Scholar Rock \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Scholar Rock \/ Inapplicable"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Chenodeoxycholic Acid","moa":"Cholesterol","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet, Film Coated","sponsorNew":"Mirum Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Mirum Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Mirum Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Chenodeoxycholic Acid","moa":"Cholesterol","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Mirum Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet, Film Coated","sponsorNew":"Mirum Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Mirum Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2023","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"GCGR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Zealand Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Danske Bank","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2023","type":"Private Placement","leadProduct":"Dasiglucagon","moa":"GCGR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Zealand Pharma \/ Danske Bank","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Danske Bank"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2022","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"GCGR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Zealand Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2022","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"GCGR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Zealand Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2023","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"GCGR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Zealand Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2024","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"GCGR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Zealand Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2023","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"GCGR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Zealand Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2022","type":"Inapplicable","leadProduct":"Dasiglucagon","moa":"GCGR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Zealand Pharma \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Zealand Pharma","sponsor":"Oberland Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Hormone","year":"2021","type":"Financing","leadProduct":"Dasiglucagon","moa":"GCGR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Zealand Pharma","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Zealand Pharma \/ Oberland Capital Management","highestDevelopmentStatusID":"10","companyTruncated":"Zealand Pharma \/ Oberland Capital Management"},{"orgOrder":0,"company":"DEKA Research & Development","sponsor":"Zealand Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Hormone","year":"2021","type":"Collaboration","leadProduct":"Dasiglucagon","moa":"GCGR","graph1":"Genetic Disease","graph2":"Phase III","graph3":"DEKA Research & Development","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"DEKA Research & Development \/ Zealand Pharma","highestDevelopmentStatusID":"10","companyTruncated":"DEKA Research & Development \/ Zealand Pharma"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Kaken Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Licensing Agreement","leadProduct":"Sebetralstat","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"KalVista Pharmaceuticals","amount2":0.02,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.02,"dosageForm":"Oral Tablet","sponsorNew":"KalVista Pharmaceuticals \/ Kaken Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"KalVista Pharmaceuticals \/ Kaken Pharmaceuticals"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Inapplicable","leadProduct":"Delpacibart Etedesiran","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Avidity Biosciences \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Avidity Biosciences \/ Inapplicable"}]

    Find Drugs for Genetic Disease in Phase III Clinical Development

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                            01

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Fitusiran

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Press Release: Two Fitusiran Phase 3 Studies Published in The Lancet and The Lancet Haematology Highlight Pote...

                            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.

                            Product Name : SAR439774

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            April 04, 2023

                            Lead Product(s) : Fitusiran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            NEJM Publishes Once-Weekly Efanesoctocog Alfa Phase 3 Data Demonstrating Its Potential to Transform the Treatm...

                            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 haemophili...

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            January 25, 2023

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Virtual Booth

                            03

                            Immunogen

                            U.S.A
                            MAGHREB PHARMA
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : Exagamglogene Autotemcel

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Vertex Pharmaceuticals

                            Deal Size : $352.0 million

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            ImmunoGen Announces a Global, Multi-Target License and Option Agreement with Vertex for the Use of ImmunoGen's...

                            Details : Vertex will have the rights to conduct research using ImmunoGen's ADC technology to discover novel targeted conditioning agents for use with gene editing. CTX001 (exagamglogene autotemcel) is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edite...

                            Product Name : CTX001

                            Product Type : Cell and Gene therapy

                            Upfront Cash : $15.0 million

                            January 03, 2023

                            Lead Product(s) : Exagamglogene Autotemcel

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Vertex Pharmaceuticals

                            Deal Size : $352.0 million

                            Deal Type : Licensing Agreement

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                            04

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Sanofi to present new clinical data reinforcing novel therapies across rare blood disorders at ASH 2022

                            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.

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            November 30, 2022

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Fitusiran

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Fitusiran Prophylaxis Reduced Bleeds by 61% in People With Hemophilia a or B, With or Without Inhibitors, Comp...

                            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.

                            Product Name : SAR439774

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            October 07, 2022

                            Lead Product(s) : Fitusiran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            Virtual Booth

                            06

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Pivotal Data Demonstrate Once-weekly Efanesoctocog Alfa Provides Superior Bleed Protection Compared to Prior F...

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

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            October 07, 2022

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Virtual Booth

                            07

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efanesoctocog Alfa Met Primary and Key Secondary Endpoints in Pivotal Study in Hemophilia a, Demonstrating Sup...

                            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.

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            September 03, 2022

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Virtual Booth

                            08

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Grants Efanesoctocog Alfa Breakthrough Therapy Designation for Hemophilia A

                            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.

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            January 06, 2022

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Virtual Booth

                            09

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Givosiran

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Data From Two Phase 3 Studies Demonstrating Fitusiran Significantly Reduced Bleeds in People with Hemophilia A...

                            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.

                            Product Name : ALN-AT3SC

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            December 14, 2021

                            Lead Product(s) : Givosiran

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            Virtual Booth

                            10

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Sanofi's Efanesoctocog Alfa Granted FDA Fast Track Designation for treatment of hemophilia A

                            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.

                            Product Name : BIVV001

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            February 18, 2021

                            Lead Product(s) : Efanesoctocog Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Data Compilation
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                            Virtual Booth