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Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2024","type":"Inapplicable","leadProduct":"Vesleteplirsen","moa":"Dystrophin expression","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2022","type":"Inapplicable","leadProduct":"Vesleteplirsen","moa":"Dystrophin expression","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2021","type":"Inapplicable","leadProduct":"Vesleteplirsen","moa":"Dystrophin expression","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Bidridistrogene Xeboparvovec","moa":"SGCB","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Bidridistrogene Xeboparvovec","moa":"SGCB","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"Bidridistrogene Xeboparvovec","moa":"SGCB","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Bidridistrogene Xeboparvovec","moa":"SGCB","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Bidridistrogene Xeboparvovec","moa":"SGCB","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide","year":"2021","type":"Inapplicable","leadProduct":"Vesleteplirsen","moa":"Dystrophin expression","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Hansa Biopharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Hansa Biopharma","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Hansa Biopharma"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"DMD*","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ F. Hoffmann-La Roche","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ F. Hoffmann-La Roche"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"SRP-9003","moa":"Beta-sarcoglycan (SGCB)","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"SRP-6004","moa":"Dysferlin (DYSF)","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"SRP-9004","moa":"Sarcoglycan alpha (SGCA)","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Arrowhead Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Licensing Agreement","leadProduct":"ARO-DUX4","moa":"DUX4 expression","graph1":"Musculoskeletal","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":11.380000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Musculoskeletal","amount2New":11.380000000000001,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Sarepta Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Hansa Biopharma","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWEDEN","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Delandistrogene Moxeparvovec","moa":"||Micro-dystrophin gene","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Sarepta Therapeutics \/ Sarepta Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Sarepta Therapeutics \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Genethon","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2020","type":"Collaboration","leadProduct":"GNT0004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Sarepta Therapeutics \/ Sarepta Therapeutics","highestDevelopmentStatusID":"6","companyTruncated":"Sarepta Therapeutics \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Arrowhead Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Musculoskeletal","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Licensing Agreement","leadProduct":"ARO-DUX4","moa":"DUX4 expression","graph1":"Musculoskeletal","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":11.380000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Musculoskeletal","amount2New":11.380000000000001,"dosageForm":"Undisclosed","sponsorNew":"Sarepta Therapeutics \/ Sarepta Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Licensing Agreement","leadProduct":"Calpain 3","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Sarepta 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Sarepta Therapeutics"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"GenEdit","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Neurology","graph2":"Preclinical","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sarepta Therapeutics \/ Sarepta Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Sarepta Therapeutics \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Lexeo Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Cardiology\/Vascular Diseases","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Financing","leadProduct":"LX2021","moa":"Undisclosed","graph1":"Cardiology\/Vascular Diseases","graph2":"Discovery","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Cardiovascular Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sarepta Therapeutics \/ Sarepta Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Sarepta Therapeutics \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Discovery","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Discovery","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sarepta Therapeutics \/ Sarepta Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Sarepta Therapeutics \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"AavantiBio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sarepta Therapeutics","amount2":0.11,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0.11,"dosageForm":"Undisclosed","sponsorNew":"Sarepta Therapeutics \/ Sarepta Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Sarepta Therapeutics \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Personalis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Undisclosed","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sarepta Therapeutics \/ Personalis","highestDevelopmentStatusID":"1","companyTruncated":"Sarepta Therapeutics \/ Personalis"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Codiak BioSciences","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Neurology","graph2":"Undisclosed","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sarepta Therapeutics \/ Sarepta Therapeutics","highestDevelopmentStatusID":"1","companyTruncated":"Sarepta Therapeutics \/ Sarepta Therapeutics"}]

Find Clinical Drug Pipeline Developments & Deals by Sarepta Therapeutics

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                          Top Deals by Deal Size (USD bn)

                          01

                          EPSC
                          Not Confirmed
                          EPSC
                          Not Confirmed

                          Details : Elevidys (delandistrogene moxeparvovec) is a single-dose, AAV-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy.

                          Product Name : Elevidys

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          May 16, 2025

                          Lead Product(s) : Delandistrogene Moxeparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          EPSC
                          Not Confirmed
                          EPSC
                          Not Confirmed

                          Details : ELEVIDYS (delandistrogene moxeparvovec) is a single-dose, AAV-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy.

                          Product Name : Elevidys

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          May 13, 2025

                          Lead Product(s) : Delandistrogene Moxeparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          EPSC
                          Not Confirmed
                          EPSC
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          May 01, 2025

                          Lead Product(s) : SRP-9005

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          EPSC
                          Not Confirmed
                          EPSC
                          Not Confirmed

                          Details : Under the license agreement, Sarepta will gain exclusive global rights to several clinical and preclinical programs, including ARO-DUX4, which is being developed as a treatment for FSHD.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : $825.0 million

                          February 10, 2025

                          Lead Product(s) : ARO-DUX4

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : Phase I

                          Recipient : Arrowhead Pharmaceuticals

                          Deal Size : $11,375.00 million

                          Deal Type : Licensing Agreement

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                          05

                          EPSC
                          Not Confirmed
                          EPSC
                          Not Confirmed

                          Details : Elevidys (delandistrogene moxeparvovec) is a single-dose gene transfer therapy, designed to to treat Duchenne muscular dystrophy by producing micro-dystrophin in skeletal muscle.

                          Product Name : Elevidys

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          January 27, 2025

                          Lead Product(s) : Delandistrogene Moxeparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          EPSC
                          Not Confirmed
                          EPSC
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 24, 2024

                          Lead Product(s) : SRP-9004

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          EPSC
                          Not Confirmed
                          EPSC
                          Not Confirmed

                          Details : SRP-9003 (bidridistrogene xeboparvovec) is an investigational gene therapy that uses the AAVrh74 vector. It is being evaluated for the treatment of limb-girdle muscular dystrophy type 2E/R4.

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 18, 2024

                          Lead Product(s) : Bidridistrogene Xeboparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          EPSC
                          Not Confirmed
                          EPSC
                          Not Confirmed

                          Details : Under the license agreement, Sarepta will gain exclusive global rights to several clinical and preclinical programs, including ARO-DUX4, which is being developed as a treatment for FSHD.

                          Product Name : Undisclosed

                          Product Type : Oligonucleotide

                          Upfront Cash : $825.0 million

                          November 26, 2024

                          Lead Product(s) : ARO-DUX4

                          Therapeutic Area : Musculoskeletal

                          Highest Development Status : Phase I/ Phase II

                          Recipient : Arrowhead Pharmaceuticals

                          Deal Size : $11,375.0 million

                          Deal Type : Licensing Agreement

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                          09

                          EPSC
                          Not Confirmed
                          EPSC
                          Not Confirmed

                          Details : Undisclosed

                          Product Name : Undisclosed

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          September 19, 2024

                          Lead Product(s) : Delandistrogene Moxeparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          EPSC
                          Not Confirmed
                          EPSC
                          Not Confirmed

                          Details : Elevidys (delandistrogene moxeparvovec) is a single-dose gene transfer therapy designed to address the underlying cause of DMD through the targeted production of micro-dystrophin in skeletal muscle.

                          Product Name : Elevidys

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          June 20, 2024

                          Lead Product(s) : Delandistrogene Moxeparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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