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Disease","country":"GERMANY","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Recombinant Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2011","type":"Inapplicable","leadProduct":"Recombinant Factor VIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bayer AG \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Bayer AG \/ Undisclosed"},{"orgOrder":0,"company":"Austin Institute for Clinical Research","sponsor":"The HydraFacial Company","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"ReGen-GF","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Austin Institute for Clinical Research","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Serum","sponsorNew":"Austin Institute for Clinical Research \/ The HydraFacial Company","highestDevelopmentStatusID":"11","companyTruncated":"Austin Institute for Clinical Research \/ The HydraFacial Company"},{"orgOrder":0,"company":"University Hospital, Lille","sponsor":"SHIRE PLC","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"IgHy10","moa":"Unknown","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"University Hospital, Lille","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University Hospital, Lille \/ SHIRE PLC","highestDevelopmentStatusID":"11","companyTruncated":"University Hospital, Lille \/ SHIRE PLC"},{"orgOrder":0,"company":"The Hospital for Sick Children","sponsor":"Canadian Hemophilia Society | Unity Health Toronto | Nationwide Children\u2019s Hospital","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Desmopressin Acetate","moa":"Vasopressin receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"The Hospital for Sick Children","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Spray","sponsorNew":"The Hospital for Sick Children \/ Canadian Hemophilia Society | Unity Health Toronto | Nationwide Children\u2019s Hospital","highestDevelopmentStatusID":"11","companyTruncated":"The Hospital for Sick Children \/ Canadian Hemophilia Society | Unity Health Toronto | Nationwide Children\u2019s Hospital"},{"orgOrder":0,"company":"Pontificia Universidad Catolica de Chile","sponsor":"Ferring Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHILE","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Desmopressin Acetate","moa":"Vasopressin receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pontificia Universidad Catolica de Chile","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pontificia Universidad Catolica de Chile \/ Ferring Pharmaceuticals","highestDevelopmentStatusID":"11","companyTruncated":"Pontificia Universidad Catolica de Chile \/ Ferring Pharmaceuticals"},{"orgOrder":0,"company":"Korea Otsuka Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SOUTH KOREA","productType":"Miscellaneous","year":"2019","type":"Inapplicable","leadProduct":"Tolvaptan","moa":"Vasopressin V2 receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Korea Otsuka Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Korea Otsuka Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Korea Otsuka Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"Voncento","moa":"von Willebrand factor | Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Chris Goss","sponsor":"Cystic Fibrosis Foundation | CF Therapeutics Development Network Coordinating Center | Medical University of South Carolina | University of Washington","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2016","type":"Inapplicable","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Chris Goss","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chris Goss \/ Cystic Fibrosis Foundation | CF Therapeutics Development Network Coordinating Center | Medical University of South Carolina | University of Washington","highestDevelopmentStatusID":"11","companyTruncated":"Chris Goss \/ Cystic Fibrosis Foundation | CF Therapeutics Development Network Coordinating Center | Medical University of South Carolina | University of Washington"},{"orgOrder":0,"company":"Versiti","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Efanesoctocog Alpha","moa":"F10","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Versiti","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Versiti \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Versiti \/ Undisclosed"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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 Alfa

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy

                            Details : Efanesoctocog Alfa is a Protein drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Hemophilia A.

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            April 24, 2025

                            Lead Product(s) : Efanesoctocog Alfa

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

                            Details : Agalsidase Beta is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Fabry Disease.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            August 31, 2023

                            Lead Product(s) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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) : alpha L Idosiduronase

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            China Post-marketing Surveillance (PMS) Study of Aldurazyme®

                            Details : Laronidase is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Mucopolysaccharidosis I.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            November 26, 2021

                            Lead Product(s) : alpha L Idosiduronase

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            AbbVie Inc

                            U.S.A
                            OTS 2025
                            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) : Pancrelipase

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Evaluate Symptoms of Exocrine Pancreatic Insufficiency in Adult Participants With Cystic Fibrosis or ...

                            Details : Pancrelipase is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            October 06, 2021

                            Lead Product(s) : Pancrelipase

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            China Post-marketing Surveillance (PMS) Study of Fabrazyme®

                            Details : Agalsidase Beta is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Fabry Disease.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            September 23, 2021

                            Lead Product(s) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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) : Imiglucerase

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type ...

                            Details : Imiglucerase is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Gaucher Disease.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            December 07, 2020

                            Lead Product(s) : Imiglucerase

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            07

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            To Assess the Glycosphingolipid Clearance and Clinical Effects of Switching to Agalsidase Beta (Fabrazyme) Ver...

                            Details : Agalsidase Beta is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Fabry Disease.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            October 30, 2019

                            Lead Product(s) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            08

                            AbbVie Inc

                            U.S.A
                            OTS 2025
                            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) : Pancrelipase

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Compare US Marketed Creon Manufactured With a Modernized Process at an Alternate Manufacturing Site...

                            Details : Pancrelipase is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            April 23, 2019

                            Lead Product(s) : Pancrelipase

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Abbvie Company Banner

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                            09

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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) : Factor VIII Fc Fusion Protein

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participa...

                            Details : Factor VIII Fc Fusion Protein is a Protein-drug Conjugate drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Hemophilia A.

                            Product Name : Undisclosed

                            Product Type : Protein-drug Conjugate

                            Upfront Cash : Inapplicable

                            March 28, 2017

                            Lead Product(s) : Factor VIII Fc Fusion Protein

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Swedish Orphan Biovitrum AB

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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                            10

                            Sanofi

                            France
                            CPhI WW Frankfurt
                            Attending
                            • 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) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsida...

                            Details : Agalsidase Beta is a Enzyme drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Fabry Disease.

                            Product Name : Undisclosed

                            Product Type : Enzyme

                            Upfront Cash : Inapplicable

                            July 26, 2012

                            Lead Product(s) : Agalsidase Beta

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            Sanofi Company Banner

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