[{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Tominersen","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chugai Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrathecal Injection","sponsorNew":"Chugai Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Chugai Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Nippon Shinyaku","sponsor":"Capricor Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell and Gene therapy","year":"2022","type":"Partnership","leadProduct":"Deramiocel","moa":"Exosome release","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Nippon Shinyaku","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Nippon Shinyaku \/ Capricor Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Nippon Shinyaku \/ Capricor Therapeutics"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Agalsidase Alfa","moa":"Globotriosylceramide","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Velaglucerase Alfa","moa":"Glucocerebroside","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Nippon Shinyaku","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Nippon Shinyaku","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Nippon Shinyaku \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Nippon Shinyaku \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2022","type":"Inapplicable","leadProduct":"Vonicog Alfa","moa":"von Willebrand factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2023","type":"Inapplicable","leadProduct":"Recombinant, Pegylated Antihemophilic Factor","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody","year":"2021","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"JCR Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody","year":"2021","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"JCR Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"JCR Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Shionogi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Zatolmilast","moa":"Phosphodiesterase 4D","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Shionogi","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Shionogi \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Shionogi \/ Inapplicable"},{"orgOrder":0,"company":"Taiho Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"TAS-205","moa":"15-LOX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Taiho Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Taiho Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Taiho Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"TAK-625","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Emixustat Hydrochloride","moa":"Retinoid isomerohydrolase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kubota Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Kubota Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Emixustat Hydrochloride","moa":"Retinoid isomerohydrolase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kubota Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Kubota Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Emixustat Hydrochloride","moa":"Retinoid isomerohydrolase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kubota Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Kubota Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Emixustat Hydrochloride","moa":"Retinoid isomerohydrolase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kubota Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Kubota Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Emixustat Hydrochloride","moa":"Retinoid isomerohydrolase","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kubota Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Kubota Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"TAK-743","moa":"Plasma kallikrein","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Arrowhead Pharmaceuticals","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Takeda Pharmaceutical \/ Arrowhead Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Arrowhead Pharmaceuticals"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2023","type":"Inapplicable","leadProduct":"Fazirsiran","moa":"SERPINA1 messenger RNA (SERPINA1 mRNA)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2021","type":"Inapplicable","leadProduct":"Immunoglobulin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2020","type":"Inapplicable","leadProduct":"TAK-667","moa":"Aurora kinase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"TAK-771","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2022","type":"Inapplicable","leadProduct":"TAK-771","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Takeda Pharmaceutical | Baxalta Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"TAK-881","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Takeda Pharmaceutical | Baxalta Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Takeda Pharmaceutical | Baxalta Pharmaceutical"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"KK8398","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Kyowa Kirin \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Kyowa Kirin \/ Inapplicable"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"JR-141","moa":"Heparan sulfate | Transferrin receptor | Dermatan sulfate","graph1":"Genetic Disease","graph2":"Phase III","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2023","type":"Inapplicable","leadProduct":"TAK-881","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Undisclosed","year":"2025","type":"Inapplicable","leadProduct":"TAK-881","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Inapplicable"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Vonicog Alfa","moa":"von Willebrand factor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"}]

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                          01

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : Vonicog Alfa is a Protein drug candidate, which is currently being evaluated in Phase III clinical studies for the treatment of von Willebrand Diseases.

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          August 19, 2025

                          Lead Product(s) : Vonicog Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          April 13, 2025

                          Lead Product(s) : KK8398

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : BPN14770 (zatolmilast) is an investigational drug that works by PDE4D inhibitor, which is used to promote the maturation of connections between neurons that are impaired in individuals with FXS.

                          Product Name : Undisclosed

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          July 18, 2024

                          Lead Product(s) : Zatolmilast

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          December 11, 2023

                          Lead Product(s) : Fazirsiran

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          October 11, 2023

                          Lead Product(s) : TAK-881

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Takeda Pharmaceutical | Baxalta Pharmaceutical

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          June 12, 2023

                          Lead Product(s) : Fazirsiran

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          January 31, 2023

                          Lead Product(s) : Recombinant, Pegylated Antihemophilic Factor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          January 10, 2023

                          Lead Product(s) : Fazirsiran

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Takeda Pharmaceutical

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Antibody

                          Upfront Cash : Inapplicable

                          October 26, 2022

                          Lead Product(s) : JR-141

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          BIOHK2025
                          Not Confirmed
                          BIOHK2025
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          October 17, 2022

                          Lead Product(s) : Vonicog Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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