[{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2019","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","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":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","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":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","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":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","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":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","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":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","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":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","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":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","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":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"BlackfinBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"BFB-101","moa":"AP4B1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BlackfinBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BlackfinBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BlackfinBio \/ Undisclosed"},{"orgOrder":0,"company":"BlackfinBio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"BFB-101","moa":"AP4B1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"BlackfinBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"BlackfinBio \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"BlackfinBio \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2018","type":"Inapplicable","leadProduct":"FLT180a","moa":"Factor IX","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Nova Laboratories","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cytotoxic Drug","year":"2018","type":"Inapplicable","leadProduct":"Hydroxyurea","moa":"Ribonucleoside-diphosphate reductase RR1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Nova Laboratories","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Nova Laboratories \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Nova Laboratories \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2020","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2022","type":"Inapplicable","leadProduct":"OTL-201","moa":"SGSH","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Orchard Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Orchard Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","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":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene 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Therapeutics \/ Syncona Limited","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Syncona Limited"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Spur Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Spur Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Syncona Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene 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\/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ReNeuron \/ Undisclosed"},{"orgOrder":0,"company":"ReNeuron","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2015","type":"Inapplicable","leadProduct":"Human Retinal Progenitor Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"ReNeuron","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Implant","sponsorNew":"ReNeuron \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"ReNeuron \/ Undisclosed"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell & Gene Therapy","year":"2015","type":"Inapplicable","leadProduct":"rAAV2tYF-CB-hRS1","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Beacon Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Beacon Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Enzyme","year":"2010","type":"Inapplicable","leadProduct":"Recombinant Human Heparan N-Sulfatase","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SHIRE PLC \/ Undisclosed"},{"orgOrder":0,"company":"SHIRE PLC","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Undisclosed","year":"2011","type":"Inapplicable","leadProduct":"Recombinant Human HNS","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"SHIRE PLC","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"SHIRE PLC \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"SHIRE PLC \/ Undisclosed"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          Aquaexpo
                          Not Confirmed
                          Aquaexpo
                          Not Confirmed

                          Details : BFB-101 is an AP4B1 gene therapy candidate, which is currently being evaluated for the treatment of Hereditary Spastic Paraplegia.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          May 07, 2025

                          Lead Product(s) : BFB-101

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          Aquaexpo
                          Not Confirmed
                          Aquaexpo
                          Not Confirmed

                          Details : BFB-101 aims to address the underlying genetic cause of hereditary spastic paraplegia, Type 47 (SPG47) by delivering a functional copy of the AP4B1 gene.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          April 28, 2025

                          Lead Product(s) : BFB-101

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          Aquaexpo
                          Not Confirmed
                          Aquaexpo
                          Not Confirmed

                          Details : FLT201 is an adeno-associated virus gene therapy candidate which is currently being investigated in the GALILEO-1 Phase 1/2 clinical trial in adults with Gaucher disease type 1.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          February 03, 2025

                          Lead Product(s) : FLT201

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Aquaexpo
                          Not Confirmed
                          Aquaexpo
                          Not Confirmed

                          Details : FLT201 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Gaucher Disease.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          August 09, 2024

                          Lead Product(s) : FLT201

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Aquaexpo
                          Not Confirmed
                          Aquaexpo
                          Not Confirmed

                          Details : FLT201 is an adeno-associated virus gene therapy candidate which is currently being investigated in the GALILEO-1 Phase 1/2 clinical trial in adults with Gaucher disease type 1.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          July 08, 2024

                          Lead Product(s) : FLT201

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Aquaexpo
                          Not Confirmed
                          Aquaexpo
                          Not Confirmed

                          Details : Through the acquisition, Syncona gains access to FLT201, a highly differentiated gene therapy candidate that delivers a novel transgene, currently in trial for Gaucher disease type 1.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : $28.3 million

                          February 20, 2024

                          Lead Product(s) : FLT201

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Syncona Limited

                          Deal Size : $28.3 million

                          Deal Type : Acquisition

                          blank

                          07

                          Aquaexpo
                          Not Confirmed
                          Aquaexpo
                          Not Confirmed

                          Details : FLT201 is an adeno-associated virus (AAV) gene therapy candidate which is currently being investigated in the GALILEO-1 Phase 1/2 clinical trial in adults with Gaucher disease type 1.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          October 04, 2023

                          Lead Product(s) : FLT201

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Aquaexpo
                          Not Confirmed
                          Aquaexpo
                          Not Confirmed

                          Details : FLT201 is an adeno-associated virus (AAV) gene therapy candidate which is currently being investigated in the GALILEO-1 Phase 1/2 clinical trial in adults with Gaucher disease type 1.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          June 26, 2023

                          Lead Product(s) : FLT201

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Aquaexpo
                          Not Confirmed
                          Aquaexpo
                          Not Confirmed

                          Details : FLT190 is designed to generate durable increases in α-Gal A levels and reduce substrate accumulation, with the aim of providing a one-time treatment that can stop disease progression and improve outcomes. It s currently being investigated for fabry dise...

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          January 19, 2023

                          Lead Product(s) : FLT190

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Aquaexpo
                          Not Confirmed
                          Aquaexpo
                          Not Confirmed

                          Details : OTL-201 is an investigational hematopoietic stem cell gene therapy being developed for the treatment of MPS-IIIA. It uses a lentiviral vector to insert a functional copy of the human SGSH gene into a patient’s hematopoietic stem cells.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          December 12, 2022

                          Lead Product(s) : OTL-201

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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