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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"}]

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : Efanesoctocog Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          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

                          Sanofi Company Banner

                          02

                          Lead Product(s) : Agalsidase Beta

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          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

                          Sanofi Company Banner

                          03

                          Lead Product(s) : alpha L Idosiduronase

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          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

                          Sanofi Company Banner

                          04

                          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

                          Abbvie Company Banner

                          05

                          Lead Product(s) : Agalsidase Beta

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          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

                          Sanofi Company Banner

                          06

                          Lead Product(s) : Imiglucerase

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          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

                          07

                          Lead Product(s) : Agalsidase Beta

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          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

                          08

                          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

                          09

                          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

                          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

                          10

                          Lead Product(s) : Agalsidase Beta

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          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