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Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Inapplicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2025","type":"Inapplicable","leadProduct":"Pegvaliase","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Garadacimab","moa":"F12a","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"CSL Behring \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"CSL Behring \/ Inapplicable"},{"orgOrder":0,"company":"GeneScience Pharmaceuticals Co Ltd","sponsor":"Children's Hospital of Fudan University | Tongji Hospital | Children's Hospital of Nanjing Medical University | Jiangxi Province Children's Hospital | Chengdu Women's and Children's Central Hospital | Shandong Provincial Hospital | Shengjing Hospital | Sh","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"CHINA","productType":"Hormone","year":"2022","type":"Inapplicable","leadProduct":"Recombinant Human Growth Hormone","moa":"Growth hormone receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"GeneScience Pharmaceuticals Co Ltd","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"GeneScience Pharmaceuticals Co Ltd \/ Children's Hospital of Fudan University | Tongji Hospital | Children's Hospital of Nanjing Medical University | Jiangxi Province Children's Hospital | Chengdu Women's and Children's Central Hospital | Shandong Provincial Hospital | Shengjing Hospital | Sh","highestDevelopmentStatusID":"11","companyTruncated":"GeneScience Pharmaceuticals Co Ltd \/ Children's Hospital of Fudan University | Tongji Hospital | Children's Hospital of Nanjing Medical University | Jiangxi Province Children's Hospital | Chengdu Women's and Children's Central Hospital | Shandong Provincial Hospital | Shengjing Hospital | Sh"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Tigermed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2024","type":"Inapplicable","leadProduct":"Anakinra","moa":"Interleukin-1 receptor","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Swedish Orphan Biovitrum AB \/ Tigermed","highestDevelopmentStatusID":"11","companyTruncated":"Swedish Orphan Biovitrum AB \/ Tigermed"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Pegvaliase","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2025","type":"Inapplicable","leadProduct":"Pegvaliase","moa":"Phenylalanine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"BioMarin Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Indiana Hemophilia &Thrombosis Center, Inc.","sponsor":"Genentech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Emicizumab","moa":"Coagulation factor IX and X","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Indiana Hemophilia &Thrombosis Center, Inc.","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Indiana Hemophilia &Thrombosis Center, Inc. \/ Genentech","highestDevelopmentStatusID":"11","companyTruncated":"Indiana Hemophilia &Thrombosis Center, Inc. \/ Genentech"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"Avalglucosidase Alfa","moa":"Glycogen","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sanofi","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Lyophilized Powder","sponsorNew":"Sanofi \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Sanofi \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Grifols International","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SPAIN","productType":"Other Large Molecule","year":"2020","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Grifols International","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Grifols International \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Grifols International \/ Inapplicable"},{"orgOrder":0,"company":"Royal College of Surgeons, Ireland","sponsor":"Imperial College London | University College Dublin | University of Limerick | Cystic Fibrosis Registry of Ireland | Gamaleya Research Institute of Epidemiology and Microbiology, Health Ministry of the Russian Federation | Medizinische Hochschule Brandenb","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Kaftrio","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Royal College of Surgeons, Ireland","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Nasal","sponsorNew":"Royal College of Surgeons, Ireland \/ Imperial College London | University College Dublin | University of Limerick | Cystic Fibrosis Registry of Ireland | Gamaleya Research Institute of Epidemiology and Microbiology, Health Ministry of the Russian Federation | Medizinische Hochschule Brandenb","highestDevelopmentStatusID":"11","companyTruncated":"Royal College of Surgeons, Ireland \/ Imperial College London | University College Dublin | University of Limerick | Cystic Fibrosis Registry of Ireland | Gamaleya Research Institute of Epidemiology and Microbiology, Health Ministry of the Russian Federation | Medizinische Hochschule Brandenb"},{"orgOrder":0,"company":"Clinic for Special Children","sponsor":"Genentech","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Risdiplam","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Clinic for Special Children","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Insert","sponsorNew":"Clinic for Special Children \/ Genentech","highestDevelopmentStatusID":"11","companyTruncated":"Clinic for Special Children \/ Genentech"},{"orgOrder":0,"company":"University of North Carolina, Chapel Hill","sponsor":"Chiesi Group","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Mannitol","moa":"Aspergillus Mannitol 2-dehydrogenase (Asperg M2DH)","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"University of North Carolina, Chapel Hill","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"University of North Carolina, Chapel Hill \/ Chiesi Group","highestDevelopmentStatusID":"11","companyTruncated":"University of North Carolina, Chapel Hill \/ Chiesi Group"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Recombinant Coagulation Factor IX","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Inapplicable"},{"orgOrder":0,"company":"University of Virginia","sponsor":"Octapharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Fibrinogen","moa":"Fibrinogen","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"University of Virginia","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"University of Virginia \/ Octapharma","highestDevelopmentStatusID":"11","companyTruncated":"University of Virginia \/ Octapharma"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Moroctocog Alfa","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Inapplicable"},{"orgOrder":0,"company":"American Thrombosis and Hemostasis Network","sponsor":"LFB USA","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Protein","year":"2020","type":"Inapplicable","leadProduct":"Coagulation Factor VIIa, Recombinant","moa":"Factor VIIa","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"American Thrombosis and Hemostasis Network","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"American Thrombosis and Hemostasis Network \/ LFB USA","highestDevelopmentStatusID":"11","companyTruncated":"American Thrombosis and Hemostasis Network \/ LFB USA"},{"orgOrder":0,"company":"Bayer AG","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"GERMANY","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Damoctocog Alfa Pegol","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Bayer AG","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Bayer AG \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Bayer AG \/ Inapplicable"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"PSI CRO","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Efanesoctocog Alfa","moa":"F8*","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Swedish Orphan Biovitrum AB","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Swedish Orphan Biovitrum AB \/ PSI CRO","highestDevelopmentStatusID":"11","companyTruncated":"Swedish Orphan Biovitrum AB \/ PSI CRO"},{"orgOrder":0,"company":"Children's Hospital Los Angeles","sponsor":"Grifols International","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"FVIII","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Children's Hospital Los Angeles","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Children's Hospital Los Angeles \/ Grifols International","highestDevelopmentStatusID":"11","companyTruncated":"Children's Hospital Los Angeles \/ Grifols International"},{"orgOrder":0,"company":"Institute of Hematology & Blood Diseases Hospital, China","sponsor":"Sinocelltech Ltd.","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"SCT800","moa":"Coagulation factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Institute of Hematology & Blood Diseases Hospital, China","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Institute of Hematology & Blood Diseases Hospital, China \/ Sinocelltech Ltd.","highestDevelopmentStatusID":"11","companyTruncated":"Institute of Hematology & Blood Diseases Hospital, China \/ Sinocelltech Ltd."},{"orgOrder":0,"company":"Austin Institute for Clinical Research","sponsor":"The HydraFacial Company","pharmaFlowCategory":"DU","therapeuticArea":"Genetic 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Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Sanofi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Efanesoctocog 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CRO"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Inapplicable","leadProduct":"Nusinersen Sodium","moa":"SMN2 pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Inapplicable"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph 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Disease","country":"U.S.A","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"IgPro","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"CSL Behring \/ Undisclosed"}]

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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 : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          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 : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          02

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          October 30, 2024

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          03

                          Lead Product(s) : Agalsidase Beta

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          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 : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          04

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          December 20, 2021

                          Lead Product(s) : Avalglucosidase Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          05

                          Lead Product(s) : alpha L Idosiduronase

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          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 : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          06

                          Details : Undisclosed

                          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 : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Abbvie Company Banner

                          07

                          Lead Product(s) : Agalsidase Beta

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          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 : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          08

                          Lead Product(s) : Alglucosidase Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          December 21, 2020

                          Lead Product(s) : Alglucosidase Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          09

                          Lead Product(s) : Imiglucerase

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          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 : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          10

                          Lead Product(s) : Agalsidase Beta

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Undisclosed

                          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 : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner