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Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and 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":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and 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":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and 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":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and 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":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"FLT190","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase 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Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Syncona Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2023","type":"Acquisition","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":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Syncona Limited","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Syncona Limited"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"FLT201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I\/ Phase 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Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Spur Therapeutics","sponsor":"Syncona Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2024","type":"Acquisition","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":"Intravenous Infusion","sponsorNew":"Spur Therapeutics \/ Syncona Limited","highestDevelopmentStatusID":"7","companyTruncated":"Spur Therapeutics \/ Syncona Limited"},{"orgOrder":0,"company":"Orchard 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II","graph3":"BlackfinBio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"BlackfinBio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"BlackfinBio \/ Inapplicable"}]

Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          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 : BFB-101

                          Product Type : Cell and 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 : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          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 : BFB-101

                          Product Type : Cell and 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 : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          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 : FLT201

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 03, 2025

                          Lead Product(s) : FLT201

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          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 : FLT201

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          August 07, 2024

                          Lead Product(s) : FLT201

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          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 : FLT201

                          Product Type : Cell and 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

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                          06

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          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 : FLT201

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          June 26, 2023

                          Lead Product(s) : FLT201

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          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 : FLT201

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          April 10, 2023

                          Lead Product(s) : FLT201

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          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 : FLT190

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          January 19, 2023

                          Lead Product(s) : FLT190

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          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 : OTL-201

                          Product Type : Cell and 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 : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          Discovery Europe
                          Not Confirmed
                          Discovery Europe
                          Not Confirmed

                          Details : Freeline intends to use the net proceeds from this offering to fund activities relating to the continued development of its product candidates, FLT180a and gene therapy platform and for other general corporate purposes.

                          Product Name : FLT180a

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          October 03, 2022

                          Lead Product(s) : Verbrinacogene Setparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Syncona Portfolio Limited

                          Deal Size : $26.1 million

                          Deal Type : Public Offering

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