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    [{"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"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            JCR Pharmaceuticals

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                            Lead Product(s) : JR-446

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            JR-446 gets US FDA Orphan Drug Status for Mucopolysaccharidosis IIIB

                            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

                            JCR Pharmaceuticals

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                            Lead Product(s) : JR-446

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Medipal Holdings Corporation

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            JCR Initiates Phase 1/2 Trial Of JR-446 for MPS Type IIIB in Japan

                            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

                            JCR Pharmaceuticals

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                            Lead Product(s) : JR-441

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            JCR Pharmaceuticals Initiates Phase I Trial of JR-441 for Mucopolysaccharidosis Type IIIA

                            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

                            JCR Pharmaceuticals

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                            Lead Product(s) : JR-441

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            JCR Completes Clinical Trial Notification for JR-441 in Mucopolysaccharidosis Type IIIA

                            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

                            JCR Pharmaceuticals

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                            Lead Product(s) : JR-441

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            U.S. FDA Grants Orphan Drug Designation to JR-441 for the Treatment of Mucopolysaccharidosis Type IIIA (MPS II...

                            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

                            JCR Pharmaceuticals

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                            Lead Product(s) : JR-441

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            JCR Pharmaceuticals Announces First Patient Dosed in a Global Phase I/II Clinical Trial of JR-441 for the Trea...

                            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

                            JCR Pharmaceuticals

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                            Japan arrow
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                            Lead Product(s) : Lepunafusp Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            JCR Pharmaceuticals Announces 52-Week Interim Data from its Global Phase I/II Study of JR-171 in Individuals w...

                            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

                            Astellas Pharma

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                            Astellas Pharma

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                            Lead Product(s) : AT845

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Astellas Announces Update on Preliminary Safety and Efficacy Data from FORTIS Study of Investigational AT845 i...

                            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

                            Astellas Pharma

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                            APGA Annual Conference
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                            Astellas Pharma

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                            Lead Product(s) : AT845

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Astellas Announces Hold Lifted by FDA on FORTIS Clinical Trial of AT845 Investigational Treatment for Adult Pa...

                            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

                            Astellas Pharma

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                            Astellas Pharma

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                            Lead Product(s) : AT845

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Astellas Announces Positive Safety Data from the FORTIS Study of AT845 in Adults with Late-Onset Pompe Disease

                            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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