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Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"RP-A501","moa":"AAV9.LAMP2B","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Rocket Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene 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Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Rocket Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic 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Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Lonza Group","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Partnership","leadProduct":"Marnetegragene Autotemcel","moa":"ITGB2","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Rocket Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rocket Pharmaceuticals \/ Lonza Group","highestDevelopmentStatusID":"7","companyTruncated":"Rocket Pharmaceuticals \/ Lonza Group"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene 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Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Rocket Pharmaceuticals \/ Inapplicable"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : The net proceeds will be used to fund the clinical development of RP-L102, which is being evaluated for the treatment of Fanconi Anemia.

                          Product Name : RP-L102

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          December 11, 2024

                          Lead Product(s) : RP-L102

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Morgan Stanley

                          Deal Size : $165.0 million

                          Deal Type : Public Offering

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                          02

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : The net proceeds will be used to fund the clinical development of RP-L102, which is being evaluated for the treatment of Fanconi Anemia.

                          Product Name : RP-L102

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          December 10, 2024

                          Lead Product(s) : RP-L102

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Morgan Stanley

                          Deal Size : $150.0 million

                          Deal Type : Public Offering

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                          03

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : RP-A501 is Rocket’s investigational gene therapy product consisting of a recombinant adeno-associated serotype 9 (AAV9) capsid, which is being evaluated for the treatment of Danon disease.

                          Product Name : RP-A501

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          September 17, 2024

                          Lead Product(s) : RP-A501

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : Kresladi (marnetegragene autotemcel) is an investigational gene therapy that delivers a functional copy of ITGB2 gene. It is being developed for treatment of severe leukocyte adhesion deficiency-I.

                          Product Name : RP-L201

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          June 28, 2024

                          Lead Product(s) : Marnetegragene Autotemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : RP-A601 is an PKP2 transgene delivering therapy candidate for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants.

                          Product Name : RP-A601

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          May 29, 2024

                          Lead Product(s) : RP-A601

                          Therapeutic Area : Cardiology/Vascular Diseases

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : Kresladi (marnetegragene autotemcel) is an investigational gene therapy delivering a functional ITGB2 gene copy for severe leukocyte adhesion deficiency-I.

                          Product Name : RP-L201

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 13, 2024

                          Lead Product(s) : Marnetegragene Autotemcel

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : RP-L102 is a lentiviral (LV) vector-based investigational gene therapy, which is being evaluated for the treatment of Fanconi Anemia, complementation group A.

                          Product Name : RP-L102

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          February 04, 2024

                          Lead Product(s) : RP-L102

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : RP-A501 is Rocket’s investigational gene therapy product for the treatment of Danon Disease and the first gene therapy for a cardiovascular condition to demonstrate safety and efficacy in clinical studies.

                          Product Name : RP-A501

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 09, 2023

                          Lead Product(s) : RP-A501

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : The proceeds will fund the development of company's pipeline of gene therapies for rare diseases, including advancement of RP-A501 in Phase 2 for Danon Disease. It consists of a recombinant AAV9 capsid containing a functional version of the human LAMP2B ...

                          Product Name : RP-A501

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          December 09, 2023

                          Lead Product(s) : RP-A501

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : J.P. Morgan

                          Deal Size : $175.0 million

                          Deal Type : Public Offering

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                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : RP-A601 is an AAV.rh74-based gene therapy candidate for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM).

                          Product Name : RP-A601

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          September 05, 2023

                          Lead Product(s) : RP-A601

                          Therapeutic Area : Cardiology/Vascular Diseases

                          Highest Development Status : IND Enabling

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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