[{"orgOrder":0,"company":"Genethon","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"GNT-003","moa":"Undisclosed","graph1":"Rare Diseases and Disorders","graph2":"Phase I\/ Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Genethon \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Genethon \/ Inapplicable"},{"orgOrder":0,"company":"Genethon","sponsor":"Hansa Biopharma","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"GNT-0003","moa":"UGT1A1","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Genethon \/ Hansa Biopharma","highestDevelopmentStatusID":"8","companyTruncated":"Genethon \/ Hansa Biopharma"},{"orgOrder":0,"company":"Genethon","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"GNT0004","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Genethon \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Genethon \/ Inapplicable"},{"orgOrder":0,"company":"Genethon","sponsor":"Sarepta Therapeutics","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":"Genethon","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Genethon \/ Sarepta Therapeutics","highestDevelopmentStatusID":"6","companyTruncated":"Genethon \/ Sarepta Therapeutics"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : GNT-0003 is being developed for crigler-najjar syndrome. The drug candidate is administered through intravenous route. Direct administration of an AAV vector carrying a normal copy of UGT1A1.

                          Product Name : GNT-0003

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          December 03, 2024

                          Lead Product(s) : GNT-0003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II

                          Sponsor : Hansa Biopharma

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : GNT0004 is an AAV8 vector-based gene therapy containing a shortened version of the DMD gene encoding dystrophin, targeting treatment for patients with Duchenne muscular dystrophy.

                          Product Name : GNT0004

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          April 23, 2024

                          Lead Product(s) : GNT0004

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase I

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : GNT-0003 is being developing a gene therapy treatment to treat patients with severe forms of CN syndrome by restoring expression of the UGT1A1 enzyme in the patient’s liver.

                          Product Name : GNT-003

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Inapplicable

                          October 01, 2023

                          Lead Product(s) : GNT-003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I/ Phase II

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          Pharma Live Expo
                          Not Confirmed
                          Pharma Live Expo
                          Not Confirmed

                          Details : CDMO’s capacity to produce AAV materials at large-scale will support Genethon and Sarepta’s project to take micro-dystrophin to clinical trials this year.

                          Product Name : GNT0004

                          Product Type : Cell and Gene therapy

                          Upfront Cash : Undisclosed

                          September 01, 2020

                          Lead Product(s) : GNT0004

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Preclinical

                          Sponsor : Sarepta Therapeutics

                          Deal Size : Undisclosed

                          Deal Type : Collaboration

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