[{"orgOrder":0,"company":"Regenxbio","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Endocrinology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Licensing Agreement","leadProduct":"Gene Therapy-AAV8","moa":"","graph1":"Endocrinology","graph2":"Undisclosed","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Endocrinology","amount2New":0,"dosageForm":"","sponsorNew":"Regenxbio \/ Ultragenyx","highestDevelopmentStatusID":"1","companyTruncated":"Regenxbio \/ Ultragenyx"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"RGX-314","moa":"VEGF","graph1":"Ophthalmology","graph2":"Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"RGX-314","moa":"VEGF","graph1":"Ophthalmology","graph2":"Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Suprachoroidal Injection","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Clearside Biomedical","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"RGX-314","moa":"VEGF","graph1":"Ophthalmology","graph2":"Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Suprachoroidal Injection","sponsorNew":"Regenxbio \/ REGENXBIO","highestDevelopmentStatusID":"8","companyTruncated":"Regenxbio \/ REGENXBIO"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"RGX-121","moa":"IDS gene","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracisternal","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Agreement","leadProduct":"NAV AAV9 vector","moa":"SMN1 gene","graph1":"Genetic Disease","graph2":"Approved","graph3":"Regenxbio","amount2":0.080000000000000002,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Intravenous Infusion","sponsorNew":"Regenxbio \/ Novartis","highestDevelopmentStatusID":"12","companyTruncated":"Regenxbio \/ Novartis"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"RGX-314","moa":"VEGF","graph1":"Ophthalmology","graph2":"Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Suprachoroidal Injection","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Not Applicable","leadProduct":"RGX-111","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebroventricular Injection","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Healthcare Royalty Management","pharmaFlowCategory":"D","therapeuticArea":"Musculoskeletal","country":"","productType":"Cell and Gene therapy","year":"2020","type":"Agreement","leadProduct":"Onasemnogene abeparvovec-xioi","moa":"SMN2 splicing","graph1":"Musculoskeletal","graph2":"Approved","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Musculoskeletal","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Regenxbio \/ Healthcare Royalty Management","highestDevelopmentStatusID":"12","companyTruncated":"Regenxbio \/ Healthcare Royalty Management"},{"orgOrder":0,"company":"Regenxbio","sponsor":"AbbVie Inc","pharmaFlowCategory":"D","therapeuticArea":"Ophthalmology","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"RGX-314","moa":"VEGF","graph1":"Ophthalmology","graph2":"Phase II","graph3":"Regenxbio","amount2":1.75,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Ophthalmology","amount2New":1.75,"dosageForm":"Suprachoroidal Injection","sponsorNew":"Regenxbio \/ AbbVie","highestDevelopmentStatusID":"8","companyTruncated":"Regenxbio \/ AbbVie"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Not Applicable","leadProduct":"RGX-314","moa":"VEGF","graph1":"Ophthalmology","graph2":"Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Suprachoroidal Injection","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"AbbVie Inc","pharmaFlowCategory":"D","therapeuticArea":"Ophthalmology","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"RGX-314","moa":"VEGF","graph1":"Ophthalmology","graph2":"Phase II","graph3":"Regenxbio","amount2":1.75,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Ophthalmology","amount2New":1.75,"dosageForm":"Suprachoroidal Injection","sponsorNew":"Regenxbio \/ AbbVie","highestDevelopmentStatusID":"8","companyTruncated":"Regenxbio \/ AbbVie"},{"orgOrder":0,"company":"Regenxbio","sponsor":"AbbVie Inc","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"RGX-314","moa":"VEGF","graph1":"Ophthalmology","graph2":"Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Regenxbio \/ Abbvie","highestDevelopmentStatusID":"10","companyTruncated":"Regenxbio \/ Abbvie"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"RGX-121","moa":"IDS gene","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"RGX-121","moa":"IDS gene","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph 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therapy","year":"2022","type":"Not Applicable","leadProduct":"RGX-111","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2022","type":"Not Applicable","leadProduct":"RGX-181","moa":"","graph1":"Genetic Disease","graph2":"IND Enabling","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"IND","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"5","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"RGX-111","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"RGX-202","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"RGX-121","moa":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"RGX-314","moa":"","graph1":"Ophthalmology","graph2":"Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Injectable\/Injection","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"RGX-202","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Cell and Gene therapy","year":"2023","type":"Not Applicable","leadProduct":"Undisclosed","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"4","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"AbbVie Inc","pharmaFlowCategory":"DU","therapeuticArea":"Ophthalmology","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"RGX-314","moa":"","graph1":"Ophthalmology","graph2":"Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Ophthalmology","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ AbbVie Inc","highestDevelopmentStatusID":"10","companyTruncated":"Regenxbio \/ AbbVie Inc"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"RGX-121","moa":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Morgan Stanley","pharmaFlowCategory":"D","therapeuticArea":"Ophthalmology","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Public Offering","leadProduct":"RGX-314","moa":"","graph1":"Ophthalmology","graph2":"Phase III","graph3":"Regenxbio","amount2":0.13,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Ophthalmology","amount2New":0.13,"dosageForm":"Injection","sponsorNew":"Regenxbio \/ Morgan Stanley","highestDevelopmentStatusID":"10","companyTruncated":"Regenxbio \/ Morgan Stanley"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"RGX-202","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"RGX-202","moa":"","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"7","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene 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Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"RGX-202","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"Regenxbio \/ Not Applicable"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"RGX-121","moa":"","graph1":"Technology","graph2":"Phase I\/II","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"","therapeuticAreaShortName":"Technology","amount2New":0.94999999999999996,"dosageForm":"","sponsorNew":"Regenxbio \/ Not Applicable","highestDevelopmentStatusID":"14","companyTruncated":"Regenxbio \/ Not Applicable"}]

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                          01

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : RGX-121 is an investigational, one-time gene therapy designed to deliver the IDS gene that encodes the iduronate-2-sulfatase enzyme (I2S). It is under development for the treatment of Hunter syndrome.

                          Brand Name : RGX-121

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          September 03, 2024

                          Lead Product(s) : RGX-121

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : RGX-202 is a cell and gene therapy drug that works by transferring the micro-dystrophin gene, It is infused intravenously for the treatment of Duchenne Muscular Dystrophy.

                          Brand Name : RGX-202

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          August 01, 2024

                          Lead Product(s) : RGX-202

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : RGX-202 is a Cell and Gene therapy drug infused intravenously for the treatment of Duchenne Muscular Dystrophy, transferring the micro-dystrophin gene.

                          Brand Name : RGX-202

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          June 24, 2024

                          Lead Product(s) : RGX-202

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : ABBV-RGX-314 is a one-time treatment for wet AMD, using the NAV® AAV8 vector to deliver a gene encoding a monoclonal anti-VEGF antibody fragment.

                          Brand Name : ABBV-RGX-314

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          March 06, 2024

                          Lead Product(s) : RGX-314

                          Therapeutic Area : Ophthalmology

                          Highest Development Status : Phase III

                          Sponsor : Morgan Stanley

                          Deal Size : $125.0 million

                          Deal Type : Public Offering

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                          05

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : RGX-202 is a recombinant AAV8 containing a transgene encoding a novel microdystrophin, which is being evaluate for the treatment of duchenne muscular dystrophy.

                          Brand Name : RGX-202

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          March 05, 2024

                          Lead Product(s) : RGX-202

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : RGX-121 is a one-time gene therapy designed to deliver the IDS gene encoding the iduronate-2-sulfatase enzyme, under development for Hunter syndrome using the NAV® AAV9 vector.

                          Brand Name : RGX-121

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          February 07, 2024

                          Lead Product(s) : RGX-121

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : RGX-202 is a recombinant AAV8 containing a transgene encoding a novel microdystrophin. It is being developed for the treatment of duchenne muscular dystrophy in patients with ages 4 to 11 years old.

                          Brand Name : RGX-202

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          February 07, 2024

                          Lead Product(s) : RGX-202

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : ABBV-RGX-314, a NAV® AAV8 vector encoding an antibody fragment, is in phase 2 trials targeting the VEGF pathway for wet AMD.

                          Brand Name : ABBV-RGX-314

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          January 16, 2024

                          Lead Product(s) : RGX-314

                          Therapeutic Area : Ophthalmology

                          Highest Development Status : Phase II

                          Sponsor : AbbVie Inc

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : RGX-202 is a Cell and Gene therapy drug infused intravenously for the treatment of Duchenne Muscular Dystrophy, transferring the micro-dystrophin gene.

                          Brand Name : RGX-202

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          November 29, 2023

                          Lead Product(s) : RGX-202

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          Pharmtech & Ingredients
                          Not Confirmed
                          Pharmtech & Ingredients
                          Not Confirmed

                          Details : Company will donate licenses to NAV® AAV8 and NAV® AAV9 from its NAV® Technology Platform to enable AMP® BGTC's development of these programs aimed at addressing ultra rare diseases i.e. Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio...

                          Brand Name : Undisclosed

                          Molecule Type : Cell and Gene therapy

                          Upfront Cash : Not Applicable

                          May 15, 2023

                          Lead Product(s) : Undisclosed

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Preclinical

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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