[{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Large Molecule","year":"2020","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Baxalta Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Baxalta Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Antihemophilic Factor (Recombinant), Plasma","moa":"Factor VIII","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Baxalta Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Baxalta Pharmaceutical \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"11","companyTruncated":"Baxalta Pharmaceutical \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"ADMA Biologics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Large Molecule","year":"2021","type":"Inapplicable","leadProduct":"Immune Globulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"ADMA Biologics","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"ADMA Biologics \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"ADMA Biologics \/ Inapplicable"},{"orgOrder":0,"company":"AbbVie Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Pancrelipase","moa":"Fatty acids | Starch | Polypeptides","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"AbbVie Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule, Delayed Release","sponsorNew":"AbbVie Inc \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"AbbVie Inc \/ Inapplicable"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pfizer Inc \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Inapplicable"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pfizer Inc \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Inapplicable"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Methotrexate","moa":"Dihydrofolate reductase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alexion Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Alexion Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Dispersible","sponsorNew":"Pfizer Inc \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Inapplicable"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pfizer Inc \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Inapplicable"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase 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Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Voxelotor","moa":"Hemoglobin HbA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"Pfizer Inc \/ 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":"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":"Injection","sponsorNew":"CSL Behring \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"CSL Behring \/ Inapplicable"},{"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 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Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Biogen \/ Inapplicable"},{"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 IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Inapplicable"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic 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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                          01

                          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

                          02

                          Details : Undisclosed

                          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 : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Abbvie Company Banner

                          03

                          BioProcess International
                          Not Confirmed
                          BioProcess International
                          Not Confirmed

                          Details : IgPro is a Antibody drug candidate, which is currently being evaluated in Phase IV clinical studies for the treatment of Immunologic Deficiency Syndromes.

                          Product Name : Undisclosed

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          July 22, 2025

                          Lead Product(s) : IgPro

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          BioProcess International
                          Not Confirmed
                          BioProcess International
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          February 04, 2025

                          Lead Product(s) : Garadacimab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          BioProcess International
                          Not Confirmed
                          BioProcess International
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          January 17, 2025

                          Lead Product(s) : Pegvaliase

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          BioProcess International
                          Not Confirmed
                          BioProcess International
                          Not Confirmed

                          Details : Spinraza (nusinersen) is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

                          Product Name : Spinraza

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          June 03, 2024

                          Lead Product(s) : Nusinersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          BioProcess International
                          Not Confirmed
                          BioProcess International
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          August 29, 2023

                          Lead Product(s) : Methotrexate

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          BioProcess International
                          Not Confirmed
                          BioProcess International
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          July 29, 2022

                          Lead Product(s) : Sacrosidase

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          BioProcess International
                          Not Confirmed
                          BioProcess International
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          February 10, 2022

                          Lead Product(s) : Risdiplam

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          BioProcess International
                          Not Confirmed
                          BioProcess International
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          February 08, 2022

                          Lead Product(s) : Voxelotor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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