[{"orgOrder":0,"company":"Hitachi Chemicals","sponsor":"Bluebird Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell and Gene therapy","year":"2020","type":"Agreement","leadProduct":"Lentiglobin","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Hitachi Chemicals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hitachi Chemicals \/ Bluebird Bio","highestDevelopmentStatusID":"7","companyTruncated":"Hitachi Chemicals \/ Bluebird Bio"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"AT845","moa":"GAA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Astellas Pharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Astellas Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"AT845","moa":"GAA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Astellas Pharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Astellas Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"AT845","moa":"GAA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Astellas Pharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Astellas Pharma \/ Inapplicable"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"AT845","moa":"GAA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Astellas Pharma","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Astellas Pharma \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Astellas Pharma \/ Inapplicable"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Lepunafusp Alpha","moa":"Dermatan sulfate hydrolytic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"JCR Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Lepunafusp Alpha","moa":"Dermatan sulfate hydrolytic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"JCR Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2021","type":"Inapplicable","leadProduct":"Lepunafusp Alpha","moa":"Dermatan sulfate hydrolytic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"JCR Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Enzyme","year":"2020","type":"Inapplicable","leadProduct":"Lepunafusp Alpha","moa":"Dermatan sulfate hydrolytic","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"JCR Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Medipal Holdings Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Large Molecule","year":"2024","type":"Inapplicable","leadProduct":"JR-446","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"JCR Pharmaceuticals \/ Medipal Holdings Corporation","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Medipal Holdings Corporation"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Large Molecule","year":"2023","type":"Inapplicable","leadProduct":"JR-441","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"JCR Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Large Molecule","year":"2024","type":"Inapplicable","leadProduct":"JR-441","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"JCR Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Large Molecule","year":"2024","type":"Inapplicable","leadProduct":"JR-441","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"JCR Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Large Molecule","year":"2023","type":"Inapplicable","leadProduct":"JR-441","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"JCR Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Medipal Holdings Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Large Molecule","year":"2024","type":"Inapplicable","leadProduct":"JR-446","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"JCR Pharmaceuticals \/ Medipal Holdings Corporation","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Medipal Holdings Corporation"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"JR-446","moa":"NAGLU\/TfR1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"JCR Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"JCR Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"JCR Pharmaceuticals \/ Inapplicable"}]

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                          01

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : JR-446 is an investigational drug being developed for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB or Sanfilippo syndrome type B).

                          Product Name : JR-446

                          Product Type : Protein

                          Upfront Cash : Inapplicable

                          May 07, 2025

                          Lead Product(s) : JR-446

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : JR-446, is a blood-brain barrier-penetrating α-N-acetylglucosaminidase, developed using JCR’s proprietary J-Brain Cargo technology, is under investigation for mucopolysaccharidosis type IIIB.

                          Product Name : JR-446

                          Product Type : Other Large Molecule

                          Upfront Cash : Inapplicable

                          December 05, 2024

                          Lead Product(s) : JR-446

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Medipal Holdings Corporation

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : JR-441 is a recombinant fusion protein of antibody fragment against the human transferrin receptor and heparan N- sulfatase, the enzyme missing or malfunctioning in subjects with MPS IIIA.

                          Product Name : JR-441

                          Product Type : Other Large Molecule

                          Upfront Cash : Inapplicable

                          October 31, 2024

                          Lead Product(s) : JR-441

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : JR-441 is a recombinant fusion protein of antibody fragment against the human transferrin receptor and heparan N- sulfatase, the enzyme missing or malfunctioning in subjects with MPS IIIA.

                          Product Name : JR-441

                          Product Type : Other Large Molecule

                          Upfront Cash : Inapplicable

                          March 09, 2024

                          Lead Product(s) : JR-441

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : JR-441 is a recombinant fusion protein of antibody fragment against the human transferrin receptor and heparan N- sulfatase, the enzyme missing or malfunctioning in subjects with Mucopolysaccharidosis Type IIIA (MPS IIIA).

                          Product Name : JR-441

                          Product Type : Other Large Molecule

                          Upfront Cash : Inapplicable

                          December 15, 2023

                          Lead Product(s) : JR-441

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : JR-441 is a recombinant fusion protein of antibody fragment against the human transferrin receptor and heparan N- sulfatase, the enzyme missing or malfunctioning in subjects with MPS IIIA.

                          Product Name : JR-441

                          Product Type : Other Large Molecule

                          Upfront Cash : Inapplicable

                          October 11, 2023

                          Lead Product(s) : JR-441

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : JR-171 (lepunafusp alfa) is a recombinant fusion protein of an antibody against the human transferrin receptor and α-L-iduronidase, the enzyme that is missing or malfunctioning in subjects with MPS I.

                          Product Name : JR-171

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          September 29, 2023

                          Lead Product(s) : Lepunafusp Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : AT845, a novel gene replacement therapy using an AAV8 vector, under a cardiac- and skeletal muscle-specific promotor, delivers a functional copy of the GAA gene, for the treatment of Late-Onset Pompe Disease (LOPD).

                          Product Name : AT845

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 22, 2023

                          Lead Product(s) : AT845

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : AT845, a novel gene replacement therapy using an AAV8 vector, under a cardiac- and skeletal muscle-specific promotor, delivers a functional copy of the GAA gene, for the treatment of Late-Onset Pompe Disease (LOPD).

                          Product Name : AT845

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          January 20, 2023

                          Lead Product(s) : AT845

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          EPS
                          Not Confirmed
                          EPS
                          Not Confirmed

                          Details : Astellas is developing AT845, a novel gene replacement therapy using an AAV8 vector, under a cardiac- and skeletal muscle-specific promotor, to deliver a functional copy of the GAA gene, for the treatment of Late-Onset Pompe Disease (LOPD).

                          Product Name : AT845

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          July 02, 2022

                          Lead Product(s) : AT845

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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