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Pharmaceutical","highestDevelopmentStatusID":"11","companyTruncated":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical"},{"orgOrder":0,"company":"Baxalta Pharmaceutical","sponsor":"Baxalta Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2017","type":"Inapplicable","leadProduct":"Human Immunoglobulin with Recombinant Hyaluronidase","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 \/ Baxalta Pharmaceutical","highestDevelopmentStatusID":"11","companyTruncated":"Baxalta Pharmaceutical \/ Baxalta Pharmaceutical"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","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"},{"orgOrder":0,"company":"ADMA Biologics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","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 \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"ADMA Biologics \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2017","type":"Inapplicable","leadProduct":"Immune Globulin","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"},{"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":"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"},{"orgOrder":0,"company":"Eli Lilly","sponsor":"Michael Rafii, MD, PhD","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody, Unconjugated","year":"2025","type":"Inapplicable","leadProduct":"Donanemab","moa":"Amyloid-beta A4 protein","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Eli Lilly","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Eli Lilly \/ Michael Rafii, MD, PhD","highestDevelopmentStatusID":"11","companyTruncated":"Eli Lilly \/ Michael Rafii, MD, PhD"},{"orgOrder":0,"company":"Paratek Pharmaceuticals","sponsor":"Paul Beringer","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibiotic","year":"2020","type":"Inapplicable","leadProduct":"Omadacycline","moa":"Bacterial 70S ribosome","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Paratek Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Paratek Pharmaceuticals \/ Paul Beringer","highestDevelopmentStatusID":"11","companyTruncated":"Paratek Pharmaceuticals \/ Paul Beringer"},{"orgOrder":0,"company":"Merck & Co","sponsor":"Joseph Kuti","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2015","type":"Inapplicable","leadProduct":"Ceftolozane Sulfate","moa":"Bacterial penicillin-binding protein","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Merck & Co","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Merck & Co \/ Joseph Kuti","highestDevelopmentStatusID":"11","companyTruncated":"Merck & Co \/ Joseph Kuti"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Ari Zimran","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2019","type":"Inapplicable","leadProduct":"Taliglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Pfizer Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Pfizer Inc \/ Ari Zimran","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Ari Zimran"},{"orgOrder":0,"company":"Pfizer Inc","sponsor":"Amy Tang","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","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":"Undisclosed","sponsorNew":"Pfizer Inc \/ Amy Tang","highestDevelopmentStatusID":"11","companyTruncated":"Pfizer Inc \/ Amy Tang"},{"orgOrder":0,"company":"Gilead Sciences","sponsor":"Ala'a Sharara","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Sofosbuvir","moa":"Hepatitis C virus NS5B RNA-dependent RNA polymerase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Gilead Sciences","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Gilead Sciences \/ Ala'a Sharara","highestDevelopmentStatusID":"11","companyTruncated":"Gilead Sciences \/ Ala'a Sharara"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Linda Randolph","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Sapropterin Hydrochloride","moa":"Phenylalanine-4-hydroxylase","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"BioMarin Pharmaceutical \/ Linda Randolph","highestDevelopmentStatusID":"11","companyTruncated":"BioMarin Pharmaceutical \/ Linda Randolph"}]

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                          01

                          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

                          02

                          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

                          03

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : Efanesoctocog Alpha 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

                          September 08, 2025

                          Lead Product(s) : Efanesoctocog Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          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, Unconjugated

                          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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                          05

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : Donanemab is a Antibody drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Down Syndrome.

                          Product Name : Undisclosed

                          Product Type : Antibody, Unconjugated

                          Upfront Cash : Inapplicable

                          April 04, 2025

                          Lead Product(s) : Donanemab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Michael Rafii, MD, PhD

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : Garadacimab is a Antibody drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Angioedemas, Hereditary.

                          Product Name : Undisclosed

                          Product Type : Antibody, Unconjugated

                          Upfront Cash : Inapplicable

                          February 04, 2025

                          Lead Product(s) : Garadacimab

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

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

                          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 : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          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

                          March 06, 2024

                          Lead Product(s) : Nusinersen Sodium

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : Methotrexate is a Other Small Molecule drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Hypophosphatasia.

                          Product Name : Undisclosed

                          Product Type : Cytotoxic Drug

                          Upfront Cash : Inapplicable

                          August 29, 2023

                          Lead Product(s) : Methotrexate

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : Mannitol is a Other Small Molecule drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Cystic Fibrosis.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          February 23, 2023

                          Lead Product(s) : Mannitol

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase IV

                          Sponsor : Chiesi Group

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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