[{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"AMT\u2011240","moa":"AOPE","graph1":"Neurology","graph2":"Preclinical","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Uniqure \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"AMT-260","moa":"Kainate receptor","graph1":"Neurology","graph2":"IND 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Inapplicable","highestDevelopmentStatusID":"15","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":"Etranacogene Dezaparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"15","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":"Etranacogene Dezaparvovec","moa":"Factor IX","graph1":"Genetic 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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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Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Inapplicable"}]

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                          01

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          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

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          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

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : AMT-162 is an investigational AAVrh10-based gene therapy that expresses a miRNA designed to knock down the expression of the mutated SOD1 protein, it is being evaluated for the the treatment of ALS.

                          Product Name : AMT-162

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          January 30, 2025

                          Lead Product(s) : AMT-162

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          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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                          05

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          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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                          06

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          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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                          07

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : AMT-260 is an AAV9 gene therapy that locally delivers miRNA to target the GRIK2 gene and suppress aberrantly expressed GluK2, which is investigated for refractory mesial temporal lobe epilepsy.

                          Product Name : AMT-260

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          November 21, 2024

                          Lead Product(s) : AMT-260

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : AMT-162 is an investigational AAVrh10-based gene therapy that expresses a miRNA designed to knock down the expression of the mutated SOD1 protein, it is being evaluated for the the treatment of ALS.

                          Product Name : AMT-162

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          October 15, 2024

                          Lead Product(s) : AMT-162

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          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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                          10

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          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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