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I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-191","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-191","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AAV5-hFIX","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase 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Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase 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Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebral Injection","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"AMT-130","moa":"Huntingtin mRNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase 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Disease","country":"NETHERLANDS","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"VO659","moa":"RNA","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Vico Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vico Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Vico Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"AMT-130","moa":"mHTT production","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrastriatal 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                          01

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

                          Details : AMT-130 lowers both normal and mutant huntingtin protein, currently being investigated for the treatment of Huntington’s disease.

                          Product Name : AMT-130

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          April 17, 2025

                          Lead Product(s) : AMT-130

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

                          Details : AMT-191 is a alpha-GLA transgene Transference cell and gene therapy drug candidate, delivered via intravenous infusion for the treatment of Fabry disease.

                          Product Name : AMT-191

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 03, 2025

                          Lead Product(s) : AMT-191

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

                          Details : The net proceeds will support the company to advance its AMT-130, which is being evaluated for the treatment of patients suffering from Huntington’s disease.

                          Product Name : AMT-130

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          January 08, 2025

                          Lead Product(s) : AMT-130

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Leerink Partners

                          Deal Size : $75.0 million

                          Deal Type : Public Offering

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                          04

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

                          Details : The net proceeds will support the company to advance its AMT-130, which is being evaluated for the treatment of patients suffering from Huntington’s disease.

                          Product Name : AMT-130

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          January 07, 2025

                          Lead Product(s) : AMT-130

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Leerink Partners

                          Deal Size : Undisclosed

                          Deal Type : Public Offering

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                          05

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

                          Details : AMT-130 is an mHTT production inhibitor cell and gene therapy drug candidate, which is currently being evaluated for the treatment of Huntington’s disease.

                          Product Name : AMT-130

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 10, 2024

                          Lead Product(s) : AMT-130

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

                          Details : AMT-191 is a alpha-GLA transgene Transference cell and gene therapy drug candidate, delivered via intravenous infusion for the treatment of Fabry disease.

                          Product Name : AMT-191

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          September 23, 2024

                          Lead Product(s) : AMT-191

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

                          Details : VO659 is an investigational allele-preferential antisense oligonucleotide (ASO) therapy, which is being evaluated for the treatment of Huntington's disease.

                          Product Name : VO659

                          Product Type : Oligonucleotide

                          Upfront Cash : Inapplicable

                          September 13, 2024

                          Lead Product(s) : VO659

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

                          Details : AMT-130 is an mHTT production inhibitor cell and gene therapy drug candidate, which is currently being evaluated for the treatment of Huntington’s disease.

                          Product Name : AMT-130

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          September 07, 2024

                          Lead Product(s) : AMT-130

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

                          Details : AMT-130 is an mHTT production inhibitor cell and gene therapy drug candidate, which is currently being evaluated for the treatment of Huntington’s disease.

                          Product Name : AMT-130

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          September 07, 2024

                          Lead Product(s) : AMT-130

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          IFT FIRST
                          Not Confirmed
                          IFT FIRST
                          Not Confirmed

                          Details : AMT-191 is a alpha-GLA transgene Transference cell and gene therapy drug candidate, delivered via intravenous infusion for the treatment of Fabry disease.

                          Product Name : AMT-191

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          August 15, 2024

                          Lead Product(s) : AMT-191

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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