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Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Arrowhead Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2024","type":"Licensing Agreement","leadProduct":"ARO-DUX4","moa":"DUX4 expression","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Sarepta Therapeutics","amount2":11.380000000000001,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":11.380000000000001,"dosageForm":"Undisclosed","sponsorNew":"Sarepta Therapeutics \/ Sarepta Therapeutics","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & 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":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","year":"2020","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":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","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":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","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":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","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":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","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":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Peptide, Unconjugated","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":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Casimersen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Casimersen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Inapplicable","leadProduct":"Casimersen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2021","type":"Inapplicable","leadProduct":"Golodirsen","moa":"Dystrophin pre-mRNA","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Solution","sponsorNew":"Sarepta Therapeutics \/ Sarepta Therapeutics","highestDevelopmentStatusID":"11","companyTruncated":"Sarepta Therapeutics \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & 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":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic 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Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","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":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","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":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & 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":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"7","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & 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":"Infusion","sponsorNew":"Sarepta Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene 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Undisclosed","highestDevelopmentStatusID":"10","companyTruncated":"Sarepta Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Licensing Agreement","leadProduct":"Calpain 3","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","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 & Gene 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Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Sarepta Therapeutics \/ Sarepta Therapeutics","highestDevelopmentStatusID":"2","companyTruncated":"Sarepta Therapeutics \/ Sarepta Therapeutics"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Lysogene","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Termination","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sarepta Therapeutics \/ Lysogene","highestDevelopmentStatusID":"9","companyTruncated":"Sarepta Therapeutics \/ Lysogene"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic 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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":"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":"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":"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"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : ARO-DM1, which is designed to reduce expression of the dystrophia myotonica protein kinase, or DMPK, gene in skeletal muscle as a potential treatment for patients with type 1 myotonic dystrophy.

                          Product Name : ARO-DM1

                          Product Type : Oligonucleotide

                          Upfront Cash : $825.0 million

                          August 13, 2025

                          Lead Product(s) : ARO-DM1

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Recipient : Arrowhead Pharmaceuticals

                          Deal Size : $11,375.0 million

                          Deal Type : Licensing Agreement

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                          02

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          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 & Gene Therapy

                          Upfront Cash : Inapplicable

                          May 16, 2025

                          Lead Product(s) : Delandistrogene Moxeparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          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 & Gene Therapy

                          Upfront Cash : Inapplicable

                          May 13, 2025

                          Lead Product(s) : Delandistrogene Moxeparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : SRP-9005 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Muscular Dystrophies, Limb-Girdle.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          May 01, 2025

                          Lead Product(s) : SRP-9005

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          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 : Genetic Disease

                          Highest Development Status : Phase I

                          Recipient : Arrowhead Pharmaceuticals

                          Deal Size : $11,375.00 million

                          Deal Type : Licensing Agreement

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                          06

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          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 & Gene Therapy

                          Upfront Cash : Inapplicable

                          January 27, 2025

                          Lead Product(s) : Delandistrogene Moxeparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          blank

                          07

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : SRP-9004 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Muscular Dystrophies, Limb-Girdle.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          December 24, 2024

                          Lead Product(s) : SRP-9004

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          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 & Gene Therapy

                          Upfront Cash : Inapplicable

                          December 18, 2024

                          Lead Product(s) : Bidridistrogene Xeboparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          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 : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Recipient : Arrowhead Pharmaceuticals

                          Deal Size : $11,375.0 million

                          Deal Type : Licensing Agreement

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                          10

                          Pharma MES 2025
                          Not Confirmed
                          Pharma MES 2025
                          Not Confirmed

                          Details : Delandistrogene Moxeparvovec is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Muscular Dystrophy, Duchenne.

                          Product Name : Undisclosed

                          Product Type : Cell & Gene Therapy

                          Upfront Cash : Inapplicable

                          September 19, 2024

                          Lead Product(s) : Delandistrogene Moxeparvovec

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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