[{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"KB105","moa":"Transglutaminase 1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Beremagene geperpavec","moa":"COL7A1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"Beremagene geperpavec","moa":"COL7A1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"KB407","moa":"CFTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Dermatology","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"KB301","moa":"COL3A1","graph1":"Dermatology","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Dermatology","amount2New":0,"dosageForm":"Intradermal Injection","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Dermatology","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"KB301","moa":"COL3A1","graph1":"Dermatology","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Dermatology","amount2New":0,"dosageForm":"Intradermal Injection","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Goldman Sachs & Co. 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LLC"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"KB407","moa":"CFTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"Beremagene Geperpavec","moa":"COL7A1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"HSV1-TGM1","moa":"Transglutaminase 1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"KB407","moa":"CFTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"Beremagene Geperpavec","moa":"COL7A1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Goldman Sachs & Co. LLC","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Public Offering","leadProduct":"Beremagene Geperpavec","moa":"COL7A1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0.20000000000000001,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20000000000000001,"dosageForm":"Topical Gel","sponsorNew":"Krystal Biotech \/ Goldman Sachs & Co. LLC","highestDevelopmentStatusID":"10","companyTruncated":"Krystal Biotech \/ Goldman Sachs & Co. LLC"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Beremagene Geperpavec","moa":"COL7A1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Beremagene Geperpavec","moa":"COL7A1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Beremagene Geperpavec","moa":"COL7A1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Beremagene Geperpavec","moa":"COL7A1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Beremagene Geperpavec","moa":"COL7A1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Topical Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Beremagene Geperpavec","moa":"COL7A1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"KB407","moa":"CFTR gene","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Inhalation Solution","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Beremagene Geperpavec","moa":"COL7A1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Beremagene Geperpavec","moa":"COL7A1","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"Beremagene Geperpavec","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Beremagene Geperpavec","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"KB407","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Beremagene Geperpavec","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph 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Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Private Placement","leadProduct":"Beremagene Geperpavec-svdt","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Krystal Biotech","amount2":0.16,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.16,"dosageForm":"Suspension","sponsorNew":"Krystal Biotech \/ Avoro Capital Advisors","highestDevelopmentStatusID":"12","companyTruncated":"Krystal Biotech \/ Avoro Capital Advisors"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Orsini Specialty Pharmacy","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Beremagene Geperpavec-svdt","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Krystal Biotech \/ Orsini Specialty Pharmacy","highestDevelopmentStatusID":"12","companyTruncated":"Krystal Biotech \/ Orsini Specialty Pharmacy"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"KB407","moa":"","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Oncology","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"KB707","moa":"","graph1":"Oncology","graph2":"IND Enabling","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Oncology","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Divestment","leadProduct":"Beremagene Geperpavec-svdt","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Krystal Biotech","amount2":0.10000000000000001,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Gel","sponsorNew":"Krystal Biotech \/ Undisclosed","highestDevelopmentStatusID":"12","companyTruncated":"Krystal Biotech \/ Undisclosed"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"KB408","moa":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"KB408","moa":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Oncology","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"KB707","moa":"","graph1":"Oncology","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Oncology","amount2New":0,"dosageForm":"Inhalation","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Beremagene Geperpavec-svdt","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Beremagene Geperpavec-svdt","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Krystal Biotech \/ Not Applicable"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Oncology","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"KB707","moa":"","graph1":"Oncology","graph2":"Phase I","graph3":"Krystal Biotech","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Oncology","amount2New":0,"dosageForm":"Solution for Nebulizer","sponsorNew":"Krystal Biotech \/ Not Applicable","highestDevelopmentStatusID":"6","companyTruncated":"Krystal Biotech \/ Not Applicable"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          euroPLX 86 Munich
                          Not Confirmed
                          euroPLX 86 Munich
                          Not Confirmed

                          Details : KB707 is a modified HSV-1 vector delivering genes for IL-12 and IL-2 to the lung, under development for advanced lung cancer.

                          Brand Name : KB707

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          April 22, 2024

                          Lead Product(s) : KB707

                          Therapeutic Area : Oncology

                          Highest Development Status : Phase I

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          euroPLX 86 Munich
                          Not Confirmed
                          euroPLX 86 Munich
                          Not Confirmed

                          Details : KB707 is a HSV-1 vector designed to deliver genes encoding IL-12 and IL-2 to the tumor microenvironment to promote systemic immune-mediated tumor clearance. It is being developed for lung cancer.

                          Brand Name : KB707

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          February 13, 2024

                          Lead Product(s) : KB707

                          Therapeutic Area : Oncology

                          Highest Development Status : Phase I

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          euroPLX 86 Munich
                          Not Confirmed
                          euroPLX 86 Munich
                          Not Confirmed

                          Details : Vyjuvek (beremagene geperpavec-svdt) is a non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds.

                          Brand Name : Vyjuvek

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          December 19, 2023

                          Lead Product(s) : Beremagene Geperpavec-svdt

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          euroPLX 86 Munich
                          Not Confirmed
                          euroPLX 86 Munich
                          Not Confirmed

                          Details : Vyjuvek (beremagene geperpavec-svdt) is a non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds.

                          Brand Name : Vyjuvek

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          November 27, 2023

                          Lead Product(s) : Beremagene Geperpavec-svdt

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          euroPLX 86 Munich
                          Not Confirmed
                          euroPLX 86 Munich
                          Not Confirmed

                          Details : KB408 is an inhaled (nebulized) formulation of the Company’s novel replication-defective, non-integrating HSV-1-based vector designed to deliver two copies of the SERPINA1 transgene, that encodes for human alpha-1 antitrypsin protein, for the treatment...

                          Brand Name : KB408

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          September 21, 2023

                          Lead Product(s) : KB408

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          euroPLX 86 Munich
                          Not Confirmed
                          euroPLX 86 Munich
                          Not Confirmed

                          Details : KB408 is an inhaled (nebulized) formulation of the Company’s novel replication-defective, non-integrating HSV-1-based vector designed to deliver two copies of the SERPINA1 transgene, that encodes for human alpha-1 antitrypsin protein, for the treatment...

                          Brand Name : KB408

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          September 05, 2023

                          Lead Product(s) : KB408

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : IND Enabling

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          euroPLX 86 Munich
                          Not Confirmed
                          euroPLX 86 Munich
                          Not Confirmed

                          Details : The Company was awarded the PRV following the FDA accelerated approval of Vyjuvek (beremagene geperpavec-svdt), , the first-ever redosable gene therapy, for the treatment of recessive or dominant dystrophic epidermolysis bullosa (DEB) for patients 6 mont...

                          Brand Name : Vyjuvek

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          August 21, 2023

                          Lead Product(s) : Beremagene Geperpavec-svdt

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Undisclosed

                          Deal Size : $100.0 million

                          Deal Type : Divestment

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                          08

                          euroPLX 86 Munich
                          Not Confirmed
                          euroPLX 86 Munich
                          Not Confirmed

                          Details : KB707 is a modified HSV-1 vector designed to deliver genes encoding both human IL-12 and IL-2 to the tumor microenvironment and promote systemic immune-mediated tumor clearance.

                          Brand Name : KB707

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          July 26, 2023

                          Lead Product(s) : KB707

                          Therapeutic Area : Oncology

                          Highest Development Status : IND Enabling

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          euroPLX 86 Munich
                          Not Confirmed
                          euroPLX 86 Munich
                          Not Confirmed

                          Details : KB407 is an investigational, redosable gene therapy designed to molecularly correct the underlying cause of CF by delivering two copies of the CFTR gene directly to the airways via nebulization.

                          Brand Name : KB407

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          July 03, 2023

                          Lead Product(s) : KB407

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          euroPLX 86 Munich
                          Not Confirmed
                          euroPLX 86 Munich
                          Not Confirmed

                          Details : Vyjuvek (beremagene geperpavec-svdt) is a non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. Vyjuvek received FDA Approval for the First-Ever Redosable Gene Therapy for DEB...

                          Brand Name : Vyjuvek

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          May 23, 2023

                          Lead Product(s) : Beremagene Geperpavec-svdt

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Orsini Specialty Pharmacy

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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