[{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"rAAV5-miHTT","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":"Intrastriatal Injection","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Etranacogene dezaparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase 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Behring","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2020","type":"Licensing Agreement","leadProduct":"Etranacogene dezaparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":2.0499999999999998,"dosageForm":"","sponsorNew":"Uniqure \/ CSL Behring","highestDevelopmentStatusID":"10","companyTruncated":"Uniqure \/ CSL Behring"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"rAAV5-miHTT","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":"Intrastriatal Injection","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"rAAV5-miHTT","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":"Stereotaxic Infusion","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"rAAV5-miHTT","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":"Stereotaxic Infusion","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Etranacogene dezaparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic 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II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrastriatal Injection","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"miRNA-based AAV Gene Therapy","moa":"","graph1":"Neurology","graph2":"Preclinical","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"rAAV5-miHTT","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":"Intrastriatal Injection","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AMT\u2011240","moa":"AOPE gene","graph1":"Neurology","graph2":"Preclinical","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"AMT-260","moa":"","graph1":"Neurology","graph2":"IND Enabling","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Etranacogene Dezaparvovec-drlb","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Apic Bio","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Licensing Agreement","leadProduct":"APB-102","moa":"","graph1":"Neurology","graph2":"IND Enabling","graph3":"Uniqure","amount2":0.059999999999999998,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Neurology","amount2New":0.059999999999999998,"dosageForm":"","sponsorNew":"Uniqure \/ uniQure","highestDevelopmentStatusID":"5","companyTruncated":"Uniqure \/ uniQure"},{"orgOrder":0,"company":"Uniqure","sponsor":"HealthCare Royalty","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2023","type":"Divestment","leadProduct":"Etranacogene Dezaparvovec-drbl","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Uniqure","amount2":0.40000000000000002,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.40000000000000002,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ HealthCare Royalty","highestDevelopmentStatusID":"12","companyTruncated":"Uniqure \/ HealthCare Royalty"},{"orgOrder":0,"company":"Uniqure","sponsor":"CSL Behring","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2023","type":"Licensing Agreement","leadProduct":"Etranacogene Dezaparvovec-drlb","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Uniqure","amount2":2.0499999999999998,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":2.0499999999999998,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ CSL Behring","highestDevelopmentStatusID":"12","companyTruncated":"Uniqure \/ CSL Behring"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"rAAV5-miHTT","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2023","type":"Not 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Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"AMT-130","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2024","type":"Not 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Applicable","leadProduct":"AMT-260","moa":"","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"AMT-130","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Uniqure \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Not Applicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2025","type":"Public Offering","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":0.080000000000000002,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Leerink Partners","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Leerink Partners"},{"orgOrder":0,"company":"Uniqure","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2025","type":"Public Offering","leadProduct":"AMT-130","moa":"mHTT production","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Infusion","sponsorNew":"Uniqure \/ Leerink Partners","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Leerink Partners"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"AMT-162","moa":"SOD1","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Intrathecal 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":"2025","type":"Inapplicable","leadProduct":"AMT-191","moa":"Alpha-GLA transgene","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"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          BioAsia
                          Not Confirmed
                          BioAsia
                          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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                          02

                          BioAsia
                          Not Confirmed
                          BioAsia
                          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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                          03

                          BioAsia
                          Not Confirmed
                          BioAsia
                          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

                          BioAsia
                          Not Confirmed
                          BioAsia
                          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

                          BioAsia
                          Not Confirmed
                          BioAsia
                          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 : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          December 10, 2024

                          Lead Product(s) : AMT-130

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          BioAsia
                          Not Confirmed
                          BioAsia
                          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 : Not Applicable

                          November 21, 2024

                          Lead Product(s) : AMT-260

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          BioAsia
                          Not Confirmed
                          BioAsia
                          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 : Not Applicable

                          October 15, 2024

                          Lead Product(s) : AMT-162

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          BioAsia
                          Not Confirmed
                          BioAsia
                          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 : Not Applicable

                          September 23, 2024

                          Lead Product(s) : AMT-191

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          BioAsia
                          Not Confirmed
                          BioAsia
                          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 : Not Applicable

                          August 15, 2024

                          Lead Product(s) : AMT-191

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          BioAsia
                          Not Confirmed
                          BioAsia
                          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 : Not Applicable

                          July 09, 2024

                          Lead Product(s) : AMT-130

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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