[{"orgOrder":0,"company":"Passage Bio","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Public Offering","leadProduct":"PBGM01","moa":"Beta-galactosidase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0.13,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.13,"dosageForm":"","sponsorNew":"Passage Bio \/ J.P. Morgan","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ J.P. Morgan"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"PBGM01","moa":"Beta-galactosidase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intra-cisterna Magna Injection","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"PBKR03","moa":"","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intra-cisterna Magna Injection\n","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"PBGM01","moa":"Beta-galactosidase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intra-cisterna Magna Injection","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Passage Bio","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Collaboration","leadProduct":"Gene therapy","moa":"","graph1":"Neurology","graph2":"Undisclosed","graph3":"Passage Bio","amount2":0.01,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Neurology","amount2New":0.01,"dosageForm":"","sponsorNew":"Passage Bio \/ Passage Bio","highestDevelopmentStatusID":"1","companyTruncated":"Passage Bio \/ Passage Bio"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"PBFT02","moa":"","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Intra-cisterna Magna Injection","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"PBGM01","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intra-cisterna Magna Injection","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"PBGM01","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intra-cisterna Magna Injection","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"University of Pennsylvania","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"PBFT02","moa":"","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Intra-cisterna Magna Injection","sponsorNew":"Passage Bio \/ University of Pennsylvania","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ University of Pennsylvania"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"PBKR03","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intra-cisterna Magna Injection","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"PBKR03","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intra-cisterna Magna Injection","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"PBGM01","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intra-Cisterna Magna Injection","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"University of Pennsylvania","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"PBKR03","moa":"Galactosylceramidase","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Passage Bio \/ University of Pennsylvania","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ University of Pennsylvania"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"PBGM01","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"University of Pennsylvania","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"PBML04","moa":"ARSA gene","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Passage Bio \/ University of Pennsylvania","highestDevelopmentStatusID":"5","companyTruncated":"Passage Bio \/ University of Pennsylvania"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"PBFT02","moa":"AAV1 gene","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Intra-cisterna Magna Injection","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"PBGM01","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"PBGM01","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"PBFT02","moa":"","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"GEMMA Biotherapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Licensing Agreement","leadProduct":"PBGM01","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0.13,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.13,"dosageForm":"","sponsorNew":"Passage Bio \/ GEMMA Biotherapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ GEMMA Biotherapeutics"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"PBFT02","moa":"","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"","sponsorNew":"Passage Bio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Not Applicable"},{"orgOrder":0,"company":"Passage Bio","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"PBFT02","moa":"GRN","graph1":"Neurology","graph2":"Phase I\/ Phase II","graph3":"Passage Bio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Injection","sponsorNew":"Passage Bio \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Passage Bio \/ Inapplicable"}]

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : PBFT02 is an adeno-associated virus delivery gene therapy. It is being evaluated for the treatment of patients with frontotemporal dementia (FTD) with granulin (GRN) mutations.

                          Product Name : PBFT02

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          January 10, 2025

                          Lead Product(s) : PBFT02

                          Therapeutic Area : Neurology

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : Through the license agreement, GEMMA will granted exclusive, worldwide rights for the development and commercialization of PBGM01 for GM1 gangliosidosis.

                          Product Name : PBGM01

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          August 01, 2024

                          Lead Product(s) : PBGM01

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : GEMMA Biotherapeutics

                          Deal Size : $134.0 million

                          Deal Type : Licensing Agreement

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                          03

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : PBFT02 is an adeno-associated virus delivery gene therapy. It is being evaluated for the treatment of patients with frontotemporal dementia (FTD) with granulin (GRN) mutations.

                          Product Name : PBFT02

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          July 16, 2024

                          Lead Product(s) : PBFT02

                          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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                          04

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : PBFT02 is an adeno-associated virus delivery gene therapy. It is being evaluated in phase 1/2 clinical trials for the treatment of patients with frontotemporal dementia (FTD) with granulin (GRN) mutations.

                          Product Name : PBFT02

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          December 20, 2023

                          Lead Product(s) : PBFT02

                          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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                          05

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1, in which patients have mutations in the GLB1 gene causing little or no residual beta-galactosidase enzyme activity and subsequent neurodegeneration.

                          Product Name : PBGM01

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          August 07, 2023

                          Lead Product(s) : PBGM01

                          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

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1, in which patients have mutations in the GLB1 gene causing little or no residual beta-galactosidase enzyme activity and subsequent neurodegeneration.

                          Product Name : PBGM01

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          December 14, 2022

                          Lead Product(s) : PBGM01

                          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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                          07

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : PBFT02 utilizes an AAV1 viral vector to deliver, through ICM administration, a functional GRN gene to patients with mutations in the gene that encodes for progranulin (PGRN).

                          Product Name : PBFT02

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          August 11, 2022

                          Lead Product(s) : PBFT02

                          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

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : PBML04, an adeno-associated virus -delivery gene therapy to treat MLD, a recessive lysosomal storage disease caused by genetic mutations that result in demyelination of fibers of central and peripheral nervous systems, leading to progressive motor and co...

                          Product Name : PBML04

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          June 08, 2022

                          Lead Product(s) : PBML04

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : University of Pennsylvania

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : The data show that the low dose of PBGM01 is well-tolerated, has a favorable safety profile and exerts a biological effect that appears to translate to improvement in developmental milestones for these children.

                          Product Name : PBGM01

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          May 18, 2022

                          Lead Product(s) : PBGM01

                          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

                          BIO CEO & Investor
                          Not Confirmed
                          BIO CEO & Investor
                          Not Confirmed

                          Details : Preclinical results, report marked improvements in both disease progression and key biomarkers in large and small animal models of Krabbe disease following a single administration of PBKR03, with no observed dose-limiting toxicities.

                          Product Name : PBKR03

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          March 29, 2022

                          Lead Product(s) : PBKR03

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : University of Pennsylvania

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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