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[{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Genethon","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Genethon strengthens its collaboration with Sarepta Therapeutics for the development of the gene therapy product GNT0004 in Duchenne muscular dystrophy","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Musculoskeletal","graph2":"Preclinical"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Muscular Dystrophy Collaboration Aims to Correct Muscle Stem Cells\u2019 DNA","therapeuticArea":"Musculoskeletal","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"January 2020","url1":"","url2":"","graph1":"Musculoskeletal","graph2":"Discovery"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Personalis","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Personalis Announces Scientific Collaboration with Sarepta Therapeutics on Immune Response to Precision Genetic Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta to Share Clinical Update from SRP-9003 Gene Therapy Trial for Limb-girdle Muscular Dystrophy Type 2E, or Beta-Sarcoglycanopathy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics Announces Positive Safety and Efficacy Data from the SRP-9001 Micro-Dystrophin Gene Therapy Trial Published in JAMA Neurology","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta's SRP-9003 Gene Therapy for LGMD2E Shows Benefits After 1 Year","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Codiak BioSciences","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$72.5 million","newsHeadline":"Sarepta and Codiak BioSciences to develop rare disease therapies","therapeuticArea":"Neurology","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"June 2020","url1":"","url2":"","graph1":"Neurology","graph2":"Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Hansa Biopharma","pharmaFlowCategory":"D","amount":"$407.5 million","upfrontCash":"$10.0 million","newsHeadline":"Hansa Biopharma Inks Agreement With Sarepta 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Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics Receives Fast Track Designation for SRP-9001 Micro-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics Announces FDA Acceptance of Casimersen (SRP-4045) NDA for Duchenne Muscular Dystrophy Amenable to Skipping Exon 45","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 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Administration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"AavantiBio","pharmaFlowCategory":"D","amount":"$107.0 million","upfrontCash":"Undisclosed","newsHeadline":"AavantiBio Launches with $107 Million Series A Financing Perceptive Advisors Including Sarepta Therapeutics","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"October 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics to Commence Dosing of the Next Study with Commercial Process Material for the SRP-9001 Gene Therapy Program for the Treatment of Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics Announces Positive Clinical Results from MOMENTUM","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics Announces Top-line Results for Part 1 of Study 102 Evaluating SRP-9001","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics Announces FDA Approval of AMONDYS 45\u2122 (casimersen) Injection for the Treatment of DMD","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"February 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta\u2019s SRP-9003 for Limb-Girdle Muscular Dystrophy Type 2E Shows Functional Improvements 2 Years After Administration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics to Share Clinical Update for 30 mg\/kg arm of MOMENTUM Study for SRP-5051","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Reports Positive Phase II Data for Next-Gen DMD Therapy SRP-5051","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics to Share Expression and Safety Results from Study 103 (ENDEAVOR) Evaluating SRP-9001","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics\u2019 SRP-9001 Demonstrates Robust Expression and Consistent Safety Profile Using Sarepta\u2019s Commercial Process Material","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Sarepta Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Sarepta Therapeutics Executes Licensing Agreement for Gene Therapy Program to Treat Limb-Girdle Muscular Dystrophy Type 2A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Preclinical"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics to Showcase Data from its Gene Therapy and RNA Platforms at World Muscle Society 2021 Virtual Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics to Initiate Part B of MOMENTUM Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Following Positive Interactions with FDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics Announces Initiation of EMBARK, a Global Pivotal Study of SRP-9001, a Gene Therapy for the Treatment of Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Lysogene","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Lysogene Announces Termination of License Agreement With Sarepta for LYS-SAF302 Program, Regaining Global Commercial Rights","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics\u2019 Gene Therapy SRP-9001 Shows Statistically Significant Functional Improvements Compared to Pre-specified Matched External Control in Part 2 of Study SRP-9001-102 for the Treatment of Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"GenEdit","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Sarepta Therapeutics and GenEdit Share Progress on Research Collaboration and Announce Agreement to Develop Gene Editing Therapeutics for Neuromuscular Diseases","therapeuticArea":"Neurology","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Neurology","graph2":"Preclinical"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics Provides Update on SRP-5051 for the Treatment of Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics to Share New Clinical 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Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Catalent Pharma Solutions","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Sarepta and Catalent Expand Strategic Manufacturing Partnership With Commercial Supply Agreement for Duchenne Muscular Dystrophy Gene Therapy Candidate","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not 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III"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy to Treat Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics Announces Update on Regulatory Review of SRP-9001","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular 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Therapy for Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics Announces Positive Data from Part B of MOMENTUM, a Phase 2 Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"}]

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            SRP-5051 (vesleteplirsen) is a next-generation peptide phosphorodiamidate morpholino oligome. It is being evaluated in phase 2 clinical trials for the treatment of patients with duchenne muscular dystrophy amenable to skipping exon 51.

            Lead Product(s): Vesleteplirsen

            Therapeutic Area: Genetic Disease Product Name: SRP-5051

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 29, 2024

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            Elevidys (delandistrogene moxeparvovec) is a single-dose gene transfer therapy for intravenous infusion designed to address the underlying cause of Duchenne muscular dystrophy through the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.

            Lead Product(s): Delandistrogene Moxeparvovec

            Therapeutic Area: Genetic Disease Product Name: Elevidys

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 30, 2023

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            In connection with this investment, the companies will explore the development of novel gene therapy candidates, including LX2021 and LX2022 for a range of cardiovascular diseases.

            Lead Product(s): LX2021

            Therapeutic Area: Cardiology/Vascular Diseases Product Name: LX2021

            Highest Development Status: Discovery Product Type: Cell and Gene therapy

            Recipient: Lexeo Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Financing August 28, 2023

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            Sarepta was awarded the PRV following U.S. FDA accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl) for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy with a confirmed mutation in the DMD gene.

            Lead Product(s): Delandistrogene Moxeparvovec-rokl

            Therapeutic Area: Genetic Disease Product Name: Elevidys

            Highest Development Status: Approved Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Undisclosed

            Deal Size: $102.0 million Upfront Cash: Undisclosed

            Deal Type: Divestment July 05, 2023

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            ELEVIDYS (delandistrogene moxeparvovec-rokl) is the first FDA approved gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne based on expression of ELEVIDYS micro-dystrophin observed in patients treated with ELEVIDYS.

            Lead Product(s): Delandistrogene Moxeparvovec-rokl

            Therapeutic Area: Genetic Disease Product Name: ELEVIDYS

            Highest Development Status: Approved Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 22, 2023

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            SRP-9001 (delandistrogene moxeparvovec) is an investigational gene transfer therapy intended to deliver SRP-9001 to muscle tissue for the targeted production of essential components of dystrophin.

            Lead Product(s): Delandistrogene Moxeparvovec

            Therapeutic Area: Genetic Disease Product Name: SRP-9001

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 24, 2023

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            SRP-9001 (delandistrogene moxeparvovec) is an investigational gene transfer therapy intended to deliver SRP-9001 to muscle tissue for the targeted production of essential components of dystrophin.

            Lead Product(s): Delandistrogene Moxeparvovec

            Therapeutic Area: Genetic Disease Product Name: SRP-9001

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 12, 2023

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            SRP-9001 (delandistrogene moxeparvovec) is an investigational gene transfer therapy intended to deliver SRP-9001 to muscle tissue for the targeted production of essential components of dystrophin.

            Lead Product(s): Delandistrogene Moxeparvovec

            Therapeutic Area: Genetic Disease Product Name: SRP-9001

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 17, 2023

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            SRP-9003 (bidridistrogene xeboparvovec) uses the MHCK7 promoter, chosen for its ability to robustly express in the heart, which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E).

            Lead Product(s): Bidridistrogene Xeboparvovec

            Therapeutic Area: Genetic Disease Product Name: SRP-9003

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 17, 2023

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            Details:

            SRP-9001 (delandistrogene moxeparvovec) is an investigational gene transfer therapy intended to deliver SRP-9001 to muscle tissue for the targeted production of essential components of dystrophin.

            Lead Product(s): Delandistrogene Moxeparvovec

            Therapeutic Area: Genetic Disease Product Name: SRP-9001

            Highest Development Status: Phase III Product Type: Cell and Gene therapy

            Recipient: Catalent Pharma Solutions

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement January 05, 2023

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