[{"orgOrder":0,"company":"Taiho Pharmaceutical","sponsor":"Undisclosed","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":"Undisclosed","sponsorNew":"Taiho Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Taiho Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","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 \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Nippon Shinyaku","sponsor":"Undisclosed","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":"Infusion","sponsorNew":"Nippon Shinyaku \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Nippon Shinyaku \/ Undisclosed"},{"orgOrder":0,"company":"Nippon Shinyaku","sponsor":"Capricor Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell & 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":"Infusion","sponsorNew":"Nippon Shinyaku \/ Capricor Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Nippon Shinyaku \/ Capricor Therapeutics"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","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":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","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":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","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 \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","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 \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","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 \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","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":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","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":"Infusion","sponsorNew":"JCR Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","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 \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"JCR Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"Undisclosed","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":"Injection","sponsorNew":"Chugai Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Chugai Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Shionogi","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","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":"Capsule","sponsorNew":"Shionogi \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Shionogi \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","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":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","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":"Tablet","sponsorNew":"Kubota Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","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":"Tablet","sponsorNew":"Kubota Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","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":"Tablet","sponsorNew":"Kubota Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","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":"Tablet","sponsorNew":"Kubota Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Kubota Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Miscellaneous","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":"Tablet","sponsorNew":"Kubota Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kubota Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","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 \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","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 \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"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":"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":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Arrowhead Pharmaceuticals","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Arrowhead Pharmaceuticals"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Antibody, Unconjugated","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 \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Undisclosed","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 \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Kyowa Kirin \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","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":"Infusion","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","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":"Undisclosed","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","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 \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","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 \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"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":"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":"Undisclosed","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 \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Undisclosed","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":"Injection","sponsorNew":"Takeda Pharmaceutical \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Takeda Pharmaceutical \/ Undisclosed"},{"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

                          FNCE
                          Not Confirmed
                          FNCE
                          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

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : KK8398 is a drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Achondroplasia.

                          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 : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          FNCE
                          Not Confirmed
                          FNCE
                          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 : Miscellaneous

                          Upfront Cash : Inapplicable

                          July 18, 2024

                          Lead Product(s) : Zatolmilast

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : Fazirsiran is a Oligonucleotide drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of alpha 1-Antitrypsin Deficiency.

                          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 : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : TAK-881 is a drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Immunologic Deficiency Syndromes.

                          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

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : Fazirsiran is a Oligonucleotide drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of alpha 1-Antitrypsin Deficiency.

                          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 : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : Recombinant, Pegylated Antihemophilic Factor is a Protein drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Hemophilia A.

                          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 : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : Fazirsiran is a Oligonucleotide drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of alpha 1-Antitrypsin Deficiency.

                          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

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : JR-141 is a Antibody drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Mucopolysaccharidosis II.

                          Product Name : Undisclosed

                          Product Type : Antibody, Unconjugated

                          Upfront Cash : Inapplicable

                          October 26, 2022

                          Lead Product(s) : JR-141

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          FNCE
                          Not Confirmed
                          FNCE
                          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

                          October 17, 2022

                          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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