[{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Syncona Portfolio Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Public Offering","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Syncona Portfolio Limited","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Syncona Portfolio Limited"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Organovo Holdings","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Series C Financing","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0.12,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Organovo Holdings","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Organovo Holdings"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Public Offering","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor 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Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Syncona Portfolio Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Public Offering","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Syncona Portfolio Limited","highestDevelopmentStatusID":"9","companyTruncated":"Spur Therapeutics \/ Syncona Portfolio Limited"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"University College London","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"Verbrinacogene Setparvovec","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Spur 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Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"AMO Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"AMO Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2020","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO Pharma","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Suspension","sponsorNew":"AMO Pharma \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"AMO Pharma \/ Undisclosed"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Tideglusib","moa":"Glycogen synthase kinase-3 beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"AMO 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Disease","amount2New":0.12,"dosageForm":"Injection","sponsorNew":"Beacon Therapeutics \/ Syncona Limited","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Syncona Limited"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"TCGX","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Series B Financing","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0.17000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17000000000000001,"dosageForm":"Injection","sponsorNew":"Beacon Therapeutics \/ TCGX","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ TCGX"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"TCGX","pharmaFlowCategory":"D","therapeuticArea":"Genetic 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Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Beacon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Beacon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2021","type":"Inapplicable","leadProduct":"rAAV2tYF-GRK1-hRPGRco","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beacon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Undisclosed"}]

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                          01

                          Florida Innovation
                          Not Confirmed
                          Florida Innovation
                          Not Confirmed

                          Details : AGTC-501 (laruparetigene zovaparvovec) is an XLRP GTPase regulator cell & gene therapy candidate, which is being evauated for the treatment of patients suffering from X-linked retinitis Pigmentosa.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          January 28, 2025

                          Lead Product(s) : Laruparetigene Zovaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          Florida Innovation
                          Not Confirmed
                          Florida Innovation
                          Not Confirmed

                          Details : AGTC-501 (laruparetigene zovaparvovec) is an XLRP GTPase regulator cell & gene therapy candidate, which is being evauated for the treatment of patients suffering from X-linked retinitis Pigmentosa.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          September 19, 2024

                          Lead Product(s) : Laruparetigene Zovaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          Florida Innovation
                          Not Confirmed
                          Florida Innovation
                          Not Confirmed

                          Details : The financing aims to advance the clinical development of Beacon's lead product AGTC-501 (laruparetigene zovaparvovec), which is being evaluated in the treatment of X-Linked Retinitis Pigmentosa.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Undisclosed

                          July 03, 2024

                          Lead Product(s) : Laruparetigene Zovaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : TCGX

                          Deal Size : $170.0 million

                          Deal Type : Series B Financing

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                          04

                          Florida Innovation
                          Not Confirmed
                          Florida Innovation
                          Not Confirmed

                          Details : AGTC-501 (laruparetigene zovaparvovec) is an XLRP GTPase regulator cell & gene therapy candidate, which is being evauated for the treatment of patients suffering from X-linked retinitis Pigmentosa.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          June 17, 2024

                          Lead Product(s) : Laruparetigene Zovaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Florida Innovation
                          Not Confirmed
                          Florida Innovation
                          Not Confirmed

                          Details : AMO-02 (tideglusib) is an investigational therapy in development for DM1 with a dual mechanism disrupting the pathogenic RNA repeat in DM1and inhibiting excess levels of the kinase GSK3β.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          May 02, 2024

                          Lead Product(s) : Tideglusib

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Florida Innovation
                          Not Confirmed
                          Florida Innovation
                          Not Confirmed

                          Details : The financing will fund Beacon's lead asset, AGTC-501, a gene therapy program in Phase II trials for the treatment of X-linked retinitis pigmentosa. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor ...

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Undisclosed

                          June 12, 2023

                          Lead Product(s) : Laruparetigene Zovaparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Syncona Limited

                          Deal Size : $120.0 million

                          Deal Type : Financing

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                          07

                          Florida Innovation
                          Not Confirmed
                          Florida Innovation
                          Not Confirmed

                          Details : AMO-02 (tideglusib), has a dual mechanism disrupting the pathogenic RNA repeat in CDM1 and inhibiting excess levels of the kinase GSK3β. It is in development for the treatment of congenital myotonic dystrophy.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          April 25, 2023

                          Lead Product(s) : Tideglusib

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Florida Innovation
                          Not Confirmed
                          Florida Innovation
                          Not Confirmed

                          Details : AMO-02 (tideglusib), has a dual mechanism disrupting the pathogenic RNA repeat in CDM1 and inhibiting excess levels of the kinase GSK3β. It is in development for the treatment of congenital myotonic dystrophy.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          February 27, 2023

                          Lead Product(s) : Tideglusib

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Florida Innovation
                          Not Confirmed
                          Florida Innovation
                          Not Confirmed

                          Details : AMO-02 (tideglusib) is in development for the treatment of congenital myotonic dystrophy and has potential for use in adult-onset myotonic dystrophy, additional CNS, neuromuscular and other orphan indications.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          December 05, 2022

                          Lead Product(s) : Tideglusib

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Florida Innovation
                          Not Confirmed
                          Florida Innovation
                          Not Confirmed

                          Details : Tideglusib is a Other Small Molecule drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Myotonic Dystrophy.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          August 13, 2021

                          Lead Product(s) : Tideglusib

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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