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    [{"orgOrder":0,"company":"Shionogi","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Zatolmilast","moa":"PDE4D","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":"Chugai Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Tominersen","moa":"Huntingtin","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":"Nippon Shinyaku","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Viltolarsen","moa":"DMD dystrophin pre-mRNA exon-53","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":"Kubota Pharmaceutical","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"JAPAN","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Emixustat Hydrochloride","moa":"RPE65","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":"RPE65","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":"RPE65","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":"RPE65","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":"RPE65","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"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Shionogi

                            Japan arrow
                            Indo Livestock Expo
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                            Shionogi

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                            Lead Product(s) : Zatolmilast

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Shionogi Updates on Zatolmilast for Fragile X Syndrome, Including Study Protocol Changes

                            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 : BPN14770

                            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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                            02

                            Kubota Pharmaceutical

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                            Kubota Pharmaceutical

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                            Indo Livestock Expo
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                            Lead Product(s) : Emixustat Hydrochloride

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Kubota Vision Announced End of Phase 3 Clinical Trial of Emixustat in Patients with Stargardt Disease

                            Details : Emixustat modulates the visual cycle by inhibiting a critical enzyme of this pathway, retinal pigment epithelium protein 65 (RPE65). The visual cycle is the process by which vitamin A is recycled in the eye; vitamin A is crucial to the visual process.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            June 23, 2022

                            Lead Product(s) : Emixustat Hydrochloride

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Takeda Pharmaceutical

                            Japan arrow
                            Indo Livestock Expo
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                            Takeda Pharmaceutical

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                            Indo Livestock Expo
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                            Lead Product(s) : Lanadelumab

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            U.S. Food and Drug Administration Accepts Takeda’s Supplemental Biologics License Application for Use of TAK...

                            Details : TAKHZYRO® (lanadelumab-flyo) Injection is a prescription medicine used to prevent attacks of hereditary angioedema (HAE) in people 12 years of age and older.

                            Product Name : Takhzyro

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            May 10, 2022

                            Lead Product(s) : Lanadelumab

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Kubota Pharmaceutical

                            Japan arrow
                            Indo Livestock Expo
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                            Kubota Pharmaceutical

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                            Indo Livestock Expo
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                            Lead Product(s) : Emixustat Hydrochloride

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Kubota Vision Announces Positive Post Hoc Analysis from Phase 3 Clinical Trial of Emixustat in Patients with S...

                            Details : Emixustat modulates visual cycle by inhibiting a critical enzyme of this pathway, retinal pigment epithelium protein 65, in analysis of Phase 3 trial it is determined that emixustat treatment resulted in a 40.8% reduction in lesion progression compared t...

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 10, 2022

                            Lead Product(s) : Emixustat Hydrochloride

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            JCR Pharmaceuticals

                            Japan arrow
                            Indo Livestock Expo
                            Not Confirmed

                            JCR Pharmaceuticals

                            Japan arrow
                            Indo Livestock Expo
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                            Lead Product(s) : Pabinafusp Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            JCR Announces First Patient Dosed in Phase 3 Global Clinical Trial of JR-141 for Treatment of MPS II (Hunter S...

                            Details : JR-141 is the first-ever approved enzyme replacement therapy (ERT) in Japan that penetrates the BBB via intravenous administration for the treatment of MPS II. JR-141 is expected to treat not only the systemic symptoms but also the neuronopathic manifest...

                            Product Name : Izcargo

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            February 15, 2022

                            Lead Product(s) : Pabinafusp Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Nippon Shinyaku

                            Japan arrow
                            Indo Livestock Expo
                            Not Confirmed

                            Nippon Shinyaku

                            Japan arrow
                            Indo Livestock Expo
                            Not Confirmed
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                            Lead Product(s) : Deramiocel

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Capricor Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Partnership

                            DEALS_DEV arrow-down

                            Nippon Shinyaku and Capricor Therapeutics enter into an Exclusive Partnership for Commercialization and Distri...

                            Details : CAP-1002 is comprised of human allogeneic cardiosphere-derived cells. Exosomes (extracellular vesicles) secreted by CAP-1002 are thought to be the mechanism of action of CAP-1002 and have been shown to reduce oxidative stress, inflammation, fibrosis.

                            Product Name : CAP-1002

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            January 25, 2022

                            Lead Product(s) : Deramiocel

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Capricor Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            07

                            JCR Pharmaceuticals

                            Japan arrow
                            Indo Livestock Expo
                            Not Confirmed

                            JCR Pharmaceuticals

                            Japan arrow
                            Indo Livestock Expo
                            Not Confirmed
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                            Lead Product(s) : Pabinafusp Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Takeda Pharmaceutical

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Takeda to Commercialize Next-Generation Hunter Syndrome Therapy Through Collaboration with JCR Pharmaceuticals

                            Details : Under the terms of the collaboration and license agreement, Takeda will exclusively commercialize JR-141, a recombinant fusion protein of an antibody against the human transferrin receptor and iduronate-2-sulfatase in the U.S. upon completion of the Pha...

                            Product Name : Izcargo

                            Product Type : Protein

                            Upfront Cash : Undisclosed

                            September 30, 2021

                            Lead Product(s) : Pabinafusp Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Takeda Pharmaceutical

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            08

                            Takeda Pharmaceutical

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                            Indo Livestock Expo
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                            Takeda Pharmaceutical

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                            Indo Livestock Expo
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                            Lead Product(s) : Recombinant Von Willebrand Factor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Takeda Data at ISTH 2021 Highlight the Benefits of Prophylaxis for Patients with Rare Bleeding Disorders

                            Details : In Europe, VEYVONDI is indicated in adults (age 18 and older) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or not indicated for the treatment of hemorrhage and treatment/prevention of surgical bleeding.

                            Product Name : Vonvendi

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            July 20, 2021

                            Lead Product(s) : Recombinant Von Willebrand Factor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Nippon Shinyaku

                            Japan arrow
                            Indo Livestock Expo
                            Not Confirmed

                            Nippon Shinyaku

                            Japan arrow
                            Indo Livestock Expo
                            Not Confirmed
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                            Lead Product(s) : Viltolarsen

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            EC Grants Orphan Drug Designation to Nippon Shinyaku's viltolarsen (NS-065/NCNP-01)

                            Details : The NDA for viltolarsen is also being reviewed under an accelerated approval pathway in the U.S. The clinical study in the US is being conducted by NS Pharma, Inc.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            August 06, 2020

                            Lead Product(s) : Viltolarsen

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Kubota Pharmaceutical

                            Japan arrow
                            Indo Livestock Expo
                            Not Confirmed

                            Kubota Pharmaceutical

                            Japan arrow
                            Indo Livestock Expo
                            Not Confirmed
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                            Lead Product(s) : Emixustat Hydrochloride

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Acucela Announces Publication Assessing the Role of Emixustat Hydrochloride in Retinal Degeneration Treatment

                            Details : This publication supports the rationale for investigating emixustat in the treatment of Stargardt disease and diabetic retinopathy.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            June 01, 2020

                            Lead Product(s) : Emixustat Hydrochloride

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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